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Articles from Muscular Dystrophy Association

Muscular Dystrophy Association and Burn Boot Camp Team Up for the 9th Annual ‘Be Their Muscle’ Philanthropic Event
Over 360 Burn Boot Camp locations in 44 states will come together to help raise money and awareness for the Muscular Dystrophy Association.
By Muscular Dystrophy Association · Via GlobeNewswire · March 31, 2025
FDA Approves Expanded Use of Eculizumab (Soliris) for Pediatric Myasthenia Gravis, Bringing New Treatment Options for Children
New York, March 10, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) welcomes the U.S. Food and Drug Administration (FDA) approval of the expanded indication of Alexion/AstraZeneca’s eculizumab (Soliris) for pediatric patients aged six years and older with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. This landmark approval makes Soliris the first and only treatment available for pediatric patients living with this debilitating neuromuscular disease. 
By Muscular Dystrophy Association · Via GlobeNewswire · March 10, 2025
Robert Califf, MD, MAAC, to Deliver Keynote Address at 2025 MDA Clinical & Scientific Conference
Following the keynote address, Dr. Califf will participate in an expert panel discussing the evolving challenges in neuromuscular disease research and care, moderated by Sharon Hesterlee, PhD, Chief Research Officer, MDA.
By Muscular Dystrophy Association · Via GlobeNewswire · February 27, 2025
Muscular Dystrophy Association Launches Nation's Largest St. Patrick’s Day Fundraising Campaign Celebrating 75 Years of Progress
Thousands of retailers will participate with in-store MDA Shamrocks pin-ups and roundups throughout February and March; and a new online limited-edition MDA Shamrocks T-shirt designed by Matt Plummer, member of the MDA Board of Directors, will be available throughout the campaign to raise awareness for rare neuromuscular diseases.
By Muscular Dystrophy Association · Via GlobeNewswire · February 18, 2025
FDA Approves New Tablet Form of Evrysdi for Treatment of Spinal Muscular Atrophy
The Muscular Dystrophy Association celebrates this new more accessible treatment option for people living with SMA.
By Muscular Dystrophy Association · Via GlobeNewswire · February 12, 2025
MDA Launches 75th Anniversary Campaign and Premieres New PSA to Shine a Light on Rare Diseases
Celebrating 75 Years of Progress, Legacy, Impact, and Momentum Leading up to Rare Disease Day on February 28
By Muscular Dystrophy Association · Via GlobeNewswire · January 28, 2025
Muscular Dystrophy Association Announces 2025 MDA National Ambassador Lily Sander
New York, Jan. 27, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) has selected Lily Sander from Charlotte, North Carolina, as its 2025 MDA National Ambassador. Sander joins MDA National Ambassador Ira Walker from Fort Lauderdale, Florida, who continues his second year representing the organization in this role. As MDA National Ambassador, Sander will participate in MDA events across the United States, raising awareness of the need for critical funding to support innovative research and care, and advocating for MDA’s legislative priorities.
By Muscular Dystrophy Association · Via GlobeNewswire · January 27, 2025
Muscular Dystrophy Association’s Legacy Awards Honor Pioneers in Clinical Research and Community Impact at 2025 MDA Clinical & Scientific Conference in March
Dr. Katherine Mathews to receive MDA Legacy Award for Achievement in Clinical Research; Donavon Decker to receive MDA Legacy Award for Community Impact in Research.
By Muscular Dystrophy Association · Via GlobeNewswire · January 21, 2025
Applications Now Open for Muscular Dystrophy Association’s College Scholarship Program for Students with Neuromuscular Conditions
MDA continues to empower the next generation of leaders by supporting higher education opportunities for students living with neuromuscular disease.
By Muscular Dystrophy Association · Via GlobeNewswire · January 13, 2025
Muscular Dystrophy Association Opens Registration for Campers and Volunteers for MDA Summer Camp 2025, Celebrating 70 Years of Transformative Summer Camp Experiences
MDA Summer Camp, offered in-person and virtually, is provided at no cost to families of children and young adults living with neuromuscular diseases. 2025 also marks the 75th Anniversary of the Muscular Dystrophy Association.
By Muscular Dystrophy Association · Via GlobeNewswire · January 7, 2025
Muscular Dystrophy Association and AFM-Téléthon Announce $510,000 Research Grant Awards Advancing Treatments for Mitochondrial Myopathies and ALS
New York, Dec. 18, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) and AFM-Téléthon (the French muscular dystrophy association) are proud to announce the awarding of two cutting-edge research grants to accelerate the development of treatments for mitochondrial myopathies and amyotrophic lateral sclerosis (ALS). These collaborative efforts represent the MDA’s commitment to funding innovative research with the potential to transform lives.
By Muscular Dystrophy Association · Via GlobeNewswire · December 18, 2024
Muscular Dystrophy Association and Coalition to Cure Calpain 3 Partner to Fund $300,000 Research Grant for Gene Therapy Development in Limb-Girdle Muscular Dystrophy
Grant Awarded to Melissa Spencer, PhD, Co-Director of the Center for Duchenne Muscular Dystrophy at UCLA.
By Muscular Dystrophy Association · Via GlobeNewswire · December 17, 2024
Muscular Dystrophy Association Celebrates USDOT Landmark Rule Supporting Safe and Accessible Air Travel for Wheelchair Users
Muscular Dystrophy Association advocates championed progress through its Accessible Air Travel Campaign throughout 2023-24.
By Muscular Dystrophy Association · Via GlobeNewswire · December 16, 2024
Muscular Dystrophy Association Announces Over $5 Million in Research Grants to Advance Neuromuscular Disease Research
14 Research Grants and 7 Development Awards for Fellowship Trainees Will Fund Critical Research Across Multiple Neuromuscular Diseases. MDA Collaboration Grants include AFM-Téléthon and Coalition to Cure Calpain 3.
By Muscular Dystrophy Association · Via GlobeNewswire · December 16, 2024
NFL Players Nyheim Hines, Raheem Blackshear, Nick Scott, and Wear Custom Cleats to Support Muscular Dystrophy Association for the NFL My Cause My Cleats Campaign
Players Use Their Platform to Spotlight MDA’s Mission and Raise Awareness to Fund Research, Care, and Advocacy for People Living with Neuromuscular Disease
By Muscular Dystrophy Association · Via GlobeNewswire · November 27, 2024
Agenda Announced for 2025 MDA Clinical & Scientific Conference Featuring Leading Speakers Dedicated to Groundbreaking Research and Clinical Achievements in Neuromuscular Disease
New York, Nov. 14, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association today announced the agenda including tracks and Chairs for the 2025 MDA Clinical & Scientific Conference being held March 16-19, 2025, in Dallas, Texas. Read the full agenda here.
By Muscular Dystrophy Association · Via GlobeNewswire · November 14, 2024
Muscular Dystrophy Association Shines a Spotlight on National Family Caregivers Awareness Month in November
New educational resources, and continued services and support are available through MDA’s Resource Center for family caregivers.
By Muscular Dystrophy Association · Via GlobeNewswire · October 31, 2024
Muscular Dystrophy Association Advocate Rhys Hoskins Honored by his Peers with Two 2024 Players Choice Awards
New York, Oct. 28, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association and the Players Trust, the charitable arm of the Major League Baseball Players Association, are thrilled to announce that Milwaukee Brewers first baseman and long-time MDA advocate Rhys Hoskins has received both the Most Valuable Philanthropist Award and the Marvin Miller Man of the Year Award in 2024 Players Choice Award voting.
By Muscular Dystrophy Association · Via GlobeNewswire · October 28, 2024
MDA Kickstart Program Receives FDA Rare Pediatric Disease Designation and Orphan Drug Designation for Congenital Myasthenic Syndrome
New York, Oct. 24, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) proudly announces that its groundbreaking MDA Kickstart program has received both an FDA Rare Pediatric Disease designation and Orphan Drug Designation for congenital myasthenic syndrome caused by CHAT (choline acetyltransferase) gene deficiency. This major milestone helps to incentivize and support the development of gene therapies targeting ultra-rare neuromuscular diseases, which often lack commercial incentives but represent high unmet medical needs.
By Muscular Dystrophy Association · Via GlobeNewswire · October 24, 2024
Muscular Dystrophy Association Amplifies National Disability Employment Awareness Month with Quest Media Panel of Industry Leaders on Wednesday, October 9 at 1 p.m. ET
Live virtual webinar event will highlight the importance of disability inclusion and provide resources for employers and jobseekers.
By Muscular Dystrophy Association · Via GlobeNewswire · September 30, 2024
MDA Awards $500,000 Grant to Accelerate ALS Research Through the HEALEY ALS Platform Trial at Massachusetts General Hospital
New York, Sept. 19, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) is proud to announce a new clinical research grant of $500,000 over three years, awarded to Sabrina Paganoni, MD, PhD, of Massachusetts General Hospital (MGH). This grant will support the HEALEY ALS Platform Trial, a pioneering initiative designed to accelerate the development of effective treatments for amyotrophic lateral sclerosis (ALS).
By Muscular Dystrophy Association · Via GlobeNewswire · September 19, 2024
MDA Kickstart Program Launches for Ultra-Rare Neuromuscular Disease Drug Development
Collaboration with UC Davis, Manufacturing Partnership with Forge Biologics
By Muscular Dystrophy Association · Via GlobeNewswire · September 9, 2024
MDA Kicks Off Muscular Dystrophy Awareness Month in September with ‘30 Days of Strength’ Campaign with Hundreds of Events to Advance Research and Care for the Neuromuscular Disease Community
Over 420 IAFF ‘Fill the Boot’ events nationwide with local fire departments over Labor Day Weekend celebrating 70 years of partnership with MDA and IAFF; NBC KSDK’s ‘Show of Strength’ Broadcast on September 1 live from Grants Farm in St. Louis from 8-10 p.m.; MDA on the Hill from September 8-10; Virtual Screening of ‘Good Bad Things’ Film from September 20 through 22. A portion of the ticket sales will benefit MDA; Access The Vote Campaign; 30 Days of Strength Fundraising Campaign
By Muscular Dystrophy Association · Via GlobeNewswire · August 26, 2024
Muscular Dystrophy Association International Association of Fire Fighters Launch 420 Fill the Boot Fundraisers in Cities Across America Labor Day Weekend for the Muscular Dystrophy Association
2024 Marks the 70th year of the MDA and IAFF partnership.
By Muscular Dystrophy Association · Via GlobeNewswire · August 19, 2024
Registration Now Open for the 2025 MDA Clinical & Scientific Conference Featuring the Latest Innovations in Neuromuscular Disease Research and Care with Global Leaders, March 16-19 in Dallas, TX
New York, July 30, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA), convener of the largest global gathering of the neuromuscular disease community, announced today that registration is now open for the 2025 MDA Clinical & Scientific Conference being held March 16-19, 2025, at the Hilton Anatole in Dallas, Texas. This milestone four-day conference will bring together renowned researchers, clinicians, academicians, advocates, and industry leaders to explore the latest advances in the field of neuromuscular disease. Early bird registration ends on December 31, 2024. To register, click here.  The 2025 MDA Clinical & Scientific Conference will feature presentations from global leaders on all aspects of pre-clinical, translational, and clinical research and care across neuromuscular diseases including amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), limb-girdle muscular dystrophy (LGMD), myasthenia gravis (MG), Charcot-Marie-Tooth disease (CMT), and many more. Session tracks will highlight topics including:
Muscular Dystrophy Association Applauds Expanded US FDA Approval of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy Patients Ages 4 and Above
Milestone Expanded Approval Brings Hope for Families Living with Duchenne Muscular Dystrophy
Muscular Dystrophy Association’s Impact to End ALS Continues with Fourth Annual Lou Gehrig Day at Major League Baseball Games
MDA community to attend and participate in more than 17 Major League Baseball games honoring Yankees first baseman the late Lou Gehrig, whose diagnosis with ALS sparked a movement to support funding for research for a cure.
MDA's Pinup & Roundup Campaigns Kick Off Across the Nation to Support Summer Camp for Kids and Young Adults Living with Neuromuscular Diseases
Support from this retail campaign provides the opportunity for over 800 kids and young adults to experience a life-changing week-long summer camp at no cost to families.
Muscular Dystrophy Association Celebrates Historic Advancements in Accessible Air Travel as Congress Passes FAA Reauthorization
Milestone Reached Following MDA Advocacy Campaign for Unprecedented Progress in Accessible Air Travel
Muscular Dystrophy Association Launches ALS Awareness Month with Public Service Announcement ‘Answering the Call’
Programming Throughout May Centers MDA Research, Care, and Advocacy
By Muscular Dystrophy Association · Via GlobeNewswire · April 29, 2024
Muscular Dystrophy Association Launches ‘Access The Vote’ Campaign to Amplify Rights of Accessible Voting
MDA Advocacy Institute: The Power of Your Vote will be held Tuesday, April 30 at 7pm ET led by Chelsey Cartwright from the League of Women Voters, sharing ways to get engaged in the electoral process.
By Muscular Dystrophy Association · Via GlobeNewswire · April 23, 2024
Muscular Dystrophy Association and Friedreich’s Ataxia Research Alliance Announce Collaborative Research Grant Using Novel Gene Editing Technology to Address Root Cause of Friedreich’s Ataxia Disease
The grant will help diversify research methods to increase the likelihood of a successful treatment for the degenerative progressive disease that affects 1 in every 40,000 people worldwide and currently has limited treatment options.
By Muscular Dystrophy Association · Via GlobeNewswire · April 17, 2024
International Association of Fire Fighters & Affiliates Launch ‘Fill the Boot’ Fundraisers Nationwide for Muscular Dystrophy Association
This is the 70th year of partnership between IAFF and MDA raising over $700 million in funds to advance care and accelerate research for MDA families living with muscular dystrophy, ALS, and related neuromuscular diseases.
By Muscular Dystrophy Association · Via GlobeNewswire · April 10, 2024
Burn Boot Camp and Muscular Dystrophy Association Team Up for 8th Annual 'Be Their Muscle' Philanthropic Event
Over 350 Burn Boot Camp locations in 41 states will come together to help raise money and awareness for Muscular Dystrophy Association.
By Muscular Dystrophy Association · Via GlobeNewswire · March 25, 2024
Muscular Dystrophy Association’s Funding of Foundational Research Leads to New FDA Approved Treatment Duvyzat (givinostat) for Duchenne Muscular Dystrophy
New York, March 21, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association’s funding of foundational research leads to new drug approval by the US Food and Drug Administration (FDA) of Duvyzat (givinostat), a histone deacetylase (HDAC) inhibitor to treat children and adolescents living with Duchenne muscular dystrophy (DMD). This drug changes gene expression in cells by altering the three-dimensional folding of DNA and has shown that it can slow DMD progression. Duvyzat (givinostat) will be made available in the United States by ITF Therapeutics.
By Muscular Dystrophy Association · Via GlobeNewswire · March 21, 2024
NFL Running Back Nyheim Hines Supports Nation's Largest St. Patrick’s Day Fundraising Campaign to Benefit Muscular Dystrophy Association
Hines continues his role as the 2024 MDA National Spokesperson and joins thousands of retail locations nationwide to support MDA Shamrocks fundraising campaign throughout March and proudly wears MDA Shamrocks t-shirt in support of online fundraising for the campaign.
By Muscular Dystrophy Association · Via GlobeNewswire · March 13, 2024
Muscular Dystrophy Association Applauds Landmark Proposal on Accessible Air Travel at The White House
MDA Advocacy #AccessibleAirTravel campaign celebrates crucial milestone for disability community.
By Muscular Dystrophy Association · Via GlobeNewswire · February 29, 2024
Brooke Eby, ALS Patient and Advocate, to deliver Keynote Address at 2024 MDA Clinical & Scientific Conference
Following the keynote address, an expert panel discusses the challenges of neuromuscular disease in the age of therapies led by Sharon Hesterlee, PhD, Chief Research Officer, MDA.
By Muscular Dystrophy Association · Via GlobeNewswire · February 15, 2024
Muscular Dystrophy Association Announces Seven Recipient Organizations of Advocacy Collaboration Grants
The Akari Foundation, All Wheels Up, Child Neurology Foundation, Cure CMD, Cure Rare Disease, Cure VCP Disease, and Christopher & Dana Reeve Foundation are recipients of MDA Advocacy Collaboration Grants.
By Muscular Dystrophy Association · Via GlobeNewswire · February 1, 2024
Muscular Dystrophy Association Announces Recipient of 2024 MDA Legacy Award for Achievement in Research, is Jeffrey Chamberlain, Ph.D., Leading Professor in Gene Therapy
New York, Jan. 30, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today announced Jeffrey Chamberlain, Ph.D., a leading professor in gene therapy focused on Duchenne muscular dystrophy (DMD) at the Institute for Stem Cell and Regenerative Medicine at the University of Washington, will receive the 2024 MDA Legacy Award for Achievement in Research, for his achievements in translational research. 
By Muscular Dystrophy Association · Via GlobeNewswire · January 30, 2024
Muscular Dystrophy Association Opens Registration for Campers and Volunteers for MDA Summer Camp 2024 to be Held at Locations Nationwide
MDA Summer Camp, held both in-person and virtually, is provided at no cost to families for children and young adults living with neuromuscular diseases.
By Muscular Dystrophy Association · Via GlobeNewswire · January 22, 2024
Muscular Dystrophy Association Announces 2024 National Ambassador Ira Walker
New York, Jan. 08, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) has selected 39-year-old Ira Walker from Fort Lauderdale, Florida, as its 2024 MDA National Ambassador. Walker joins 16-year-old National Ambassador Leah Zelaya from Brooklyn, New York, who continues her second year representing the organization. As an MDA National Ambassador, Walker will participate in MDA events across the United States, raising awareness of the need for critical funding to support innovative research and care, and advocating for MDA legislative priorities.
By Muscular Dystrophy Association · Via GlobeNewswire · January 8, 2024
Muscular Dystrophy Association Announces Acosta Group’s Historic $100 Million Fundraising Milestone
38-year collaboration drives funding to support critical MDA research, care, and advocacy.
By Muscular Dystrophy Association · Via GlobeNewswire · December 21, 2023
Muscular Dystrophy Association Launches Holiday Retail Campaign in Thousands of Retail Locations Nationwide
New York, Nov. 01, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today launched its largest Holiday Retail Campaign to fundraise in thousands of retail locations nationwide from November through January 2024. For over a decade, retailers including GPM Investments, Circle K, Graham C-Stores, Graham Enterprises, Florida Turnpike Services and Sam’s Mart form a nationwide network to raise funds to accelerate research, advance care, and advocate to empower families living with muscular dystrophy, ALS, and related neuromuscular diseases. Retailers invite customers to participate in the MDA Holiday campaign by donating at checkout or rounding up their purchase. Donations made by customers will be displayed on special MDA Holiday pinups in each location, showing support for MDA families. Donations to this year's holiday campaign may also be made directly online here.
By Muscular Dystrophy Association · Via GlobeNewswire · November 1, 2023
Muscular Dystrophy Association Announces Collaboration with Mitsubishi Tanabe Pharma America for Remote Digital Technology Research Study for Amyotrophic Lateral Sclerosis (ALS)
New York, Oct. 30, 2023 (GLOBE NEWSWIRE) -- Muscular Dystrophy Association (MDA) and Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced their collaboration for a remote observational research study focusing on amyotrophic lateral sclerosis (ALS). The newly developed research study, ALS Go-Digital, is designed to pair digital data collected remotely with longitudinal clinical data. The study began recruiting participants in September 2023.
By Muscular Dystrophy Association · Via GlobeNewswire · October 30, 2023
Muscular Dystrophy Association-Supported Drug AGAMREE® (vamorolone) Approved for the Treatment of Duchenne Muscular Dystrophy by the FDA
New York, Oct. 26, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of AGAMREE® (vamorolone), a structurally unique steroidal anti-inflammatory drug to treat children and adolescents living with Duchenne muscular dystrophy (DMD). This multi-functional drug shows potent inhibition of pro-inflammatory NFkB pathways via high-affinity binding to the glucocorticoid receptor, high-affinity antagonism for the mineralocorticoid receptor, and membrane stabilization properties. This novel therapy, supported in part by MDA Venture Philanthropy, demonstrates similar efficacy to traditional corticosteroids with reduced negative downstream impacts or side effects. Vamorolone will be made available in the United States by Catalyst Pharmaceuticals.
By Muscular Dystrophy Association · Via GlobeNewswire · October 26, 2023
Muscular Dystrophy Association Celebrates FDA Approval of ZILBRYSQ® zilucoplan for the Treatment of Generalized Myasthenia Gravis (gMG) in Adult Patients
New York, Oct. 18, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of ZILBRYSQ® zilucoplan for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody-positive. Zilucoplan is the first once-daily subcutaneous, targeted C5 complement inhibitor for gMG, and it is the only once-daily gMG-target therapy for self-administration. Zilucoplan will be made available in the United States by UCB.
By Muscular Dystrophy Association · Via GlobeNewswire · October 18, 2023
Muscular Dystrophy Association Celebrates FDA Approval of Amicus Therapeutics’ Pombiliti + Opfolda for Treatment of Pompe Disease
New York, Sept. 28, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules for adults living with late-onset Pompe disease (LOPD). This two-component therapy is indicated for adults weighing ≥40 kg who are not improving on their current enzyme replacement therapy (ERT), and is the first and only two-component therapy for elibible adults with LOPD. Pombiliti™ + Opfolda™ will be made available and marketed in the United States by Amicus Therapeutics.  
By Muscular Dystrophy Association · Via GlobeNewswire · September 28, 2023
Muscular Dystrophy Association Announces Research Grants Totaling $8.4 Million
New York, Sept. 13, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) announced today the awarding of 37 new grants with a total funding commitment of $8.4 million toward neuromuscular disease research. The newly funded projects aim to advance research discoveries and new therapy development in multiple areas including amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), Duchenne muscular dystrophy (DMD), Emery-Dreifuss muscular dystrophy (EDMD), limb-girdle muscular dystrophy (LGMD), spinal muscular atrophy (SMA), myotonic dystrophy (DM), and many more.
By Muscular Dystrophy Association · Via GlobeNewswire · September 13, 2023
Muscular Dystrophy Association Kicks Off 30 Days of Strength Campaign for National Muscular Dystrophy Awareness Month in September
MDA Board approves 37 research grants totaling $8.4 million toward neuromuscular disease research to advance research discoveries and new therapy development in amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), Duchenne muscular dystrophy (DMD), Emory-Dreifuss muscular dystrophy (EDMD), limb-girdle muscular dystrophy (LGMD), spinal muscular atrophy (SMA), myotonic dystrophy (DM), and many more.
By Muscular Dystrophy Association · Via GlobeNewswire · August 28, 2023
Muscular Dystrophy Association International Association of Fire Fighters Launch 420 Fill the Boot Fundraisers in Cities Across America Labor Day Weekend for the Muscular Dystrophy Association
New York, Aug. 21, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) in partnership with the International Association of Fire Fighters (IAFF) today kick off more than 420 Fill the Boot events to raise lifesaving funds throughout Labor Day weekend in over 40 states. The funds raised by more than 339,000 IAFF members and their affiliates across the country go towards MDA's mission for scientific and clinical research and care for families living with muscular dystrophy, ALS (also known as Lou Gehrig’s disease), and related neuromuscular diseases.
By Muscular Dystrophy Association · Via GlobeNewswire · August 21, 2023
Registration Now Open for 2024 MDA Clinical & Scientific Conference, to Showcase Cutting Edge Research Advancements and Clinical Achievements in Neuromuscular Disease
Global leaders will gather at the annual MDA Clinical & Scientific Conference March 3-6, 2024. Presentations will be held in-person in Orlando, Florida and available virtually.
By Muscular Dystrophy Association · Via GlobeNewswire · August 9, 2023
Muscular Dystrophy Association Celebrates FDA Approval of UCB’s RYSTIGGO for the Treatment of Generalized Myasthenia Gravis
New York, June 27, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of RYSTIGGO (rozanolixizumab-noli) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. RYSTIGGO will be made available and marketed in the United States by UCB.
Muscular Dystrophy Association Celebrates FDA Approval of Sarepta Therapeutics’ ELEVIDYS for Treatment of Duchenne Muscular Dystrophy
New York, June 22, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. ELEVIDYS is the first gene therapy for DMD, and is designed to target the underlying cause of the disease. ELEVIDYS will be made available and marketed in the United States by Sarepta Therapeutics.  DMD is caused by mutations in the dystrophin gene (DMD) on the X chromosome that result in little or no production of dystrophin, a protein essential to keeping muscle cells intact. People with DMD experience progressive muscle degeneration and weakness. Over time, DMD becomes fatal as muscle degeneration disrupts lung and heart function.
Muscular Dystrophy Association Celebrates FDA Approval of argenx’s Vyvgart Hytrulo Injection for Treatment of Generalized Myasthenia Gravis
New York, June 20, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of subcutaneous (SC) Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc),for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. Vyvgart Hytrulo is the first FDA-approved SC injectable for gMG and will be made available and marketed in the United States by argenx.
CureDuchenne, Muscular Dystrophy Association, and Parent Project Muscular Dystrophy Announce Collaborative Project to Focus on Re-Dosing Gene Therapy in Duchenne Muscular Dystrophy
New York, June 15, 2023 (GLOBE NEWSWIRE) -- June 15, 2023 -- CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD), today announced a collaborative clinical trial grant to test repurposing of the FDA-approved drug Vyvgart (developed by argenx), for its potential ability to reduce antibodies to adeno-associated virus (AAV) in Duchenne muscular dystrophy (DMD) patients. The three organizations provided $100,000 each to sponsor a one-year clinical study led by investigator Barry Byrne, M.D., Ph.D., Chief Medical Advisor, MDA and Director of the Powell Gene Therapy Center at the University of Florida to assess the safety and efficacy of efgartigimod alfa-fcab (Vyvgart) to lower AAV antibodies precluding DMD patients from gene therapy. Vyvgart is an approved drug currently used to treat autoimmune diseases by reducing overall levels of circulating IgG antibodies. It has proven effective in reducing pathogenic acetylcholine receptor antibodies present in myasthenia gravis (MG). The study will investigate whether Vyvgart has the ability to reduce AAV antibodies to levels compatible with safe and effective delivery of gene therapies in DMD patients. Positive results from this study may be applicable to the translation of AAV gene therapies for other neuromuscular diseases.
MDA Launches Pinup & Roundup Campaigns at Thousands of Retail Partner Locations Nationwide to Fund Summer Camp for Kids and Young Adults 8-17 Living with Muscular Dystrophy & Related Neuromuscular Diseases
New York, May 25, 2023 (GLOBE NEWSWIRE) -- Muscular Dystrophy Association (MDA) today announced over 1,000 retail partner locations nationwide are participating with customer pinup, roundup or giving campaigns, now through October. Partners include Alaska Commercial Company, Albertsons Companies Foundation, American Furniture Warehouse, Circle K, Food Depot, Green Valley Grocery, Gordon Foods, Graham C-Stores, Graham Enterprises, Jon’s Fresh Marketplace, Marden’s Surplus, Mar-Val Food Stores, Pete’s Market, SavOn Convenience Stores, Vallarta Supermarkets, and Walgreens. To learn more about the MDA Summer Camp retail program and how to get involved, visit https://bit.ly/SummerRetail.
Muscular Dystrophy Association Announces Gene Therapy Support Network for Families Living with Neuromuscular Disease
MDA Gene Therapy Support Network offers guidance on MDA Care Centers, resources, and educational programming beginning with MDA Virtual Learning: Gene Therapy 101 webinar to be held Thursday, June 15, 4-5:30pm ET.
ABC Eyewitness News Anchor Bill Ritter to Host Muscular Dystrophy Association’s 23rd Annual Wings Over Wall Street Gala to Benefit ALS Research, Honoring Dr. Angela Genge, Lisa Galante, R.N., Susan Jaycox
New York, May 18, 2023 (GLOBE NEWSWIRE) -- Muscular Dystrophy Association's (MDA) 23rd annual Wings Over Wall Street (WOWS) gala, will be held Thursday, June 1 at 6pm at The Edison Ballroom in New York City. Funds raised will go towards MDA's ALS research, to keep the pipeline of promising therapeutics moving forward for people living with amyotrophic lateral sclerosis (ALS) aka Lou Gehrig's disease. ABC Eyewitness News Anchor, Bill Ritter, will once again host the event honoring Angela Genge, M.D., FRCP(C), eMBA, the first international recipient of the Diamond Award, Lisa Galante, R.N., NEALS Clinical Research Institute Ambassador the recipient of the Spirit Award, and Susan Jaycox, recipient of the Beier Award, for their outstanding contributions to the mission. Register to attend the event here.
Muscular Dystrophy Association Tribute Awards in Washington D.C. Honor Diana X. Bharucha-Goebel, M.D., R. Rodney Howell, M.D., Paul Robertson, Jeni Stepanek, Ph.D., Janet Woodcock, M.D.
MDA Tribute Tour in Washington, D.C. includes raising awareness with Major League Baseball for Lou Gehrig Day with the Washington Nationals vs. Phillies June 2.
Muscular Dystrophy Association Celebrates FDA Approval of Biogen’s Qalsody for Treatment of SOD1-ALS
New York, April 25, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) accelerated approval of Qalsody (tofersen), for the treatment of amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) associated with mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). Qalsody is the fourth approved therapy to treat a form of ALS and the first therapy to target a genetic cause of ALS. Qalsody will be made available and marketed in the United States by Biogen Inc. 
By Muscular Dystrophy Association · Via GlobeNewswire · April 25, 2023
International Association of Fire Fighters & Affiliates Launch ‘Fill the Boot’ Fundraisers Nationwide for Muscular Dystrophy Association
For nearly 70 years, IAFF 'Fill the Boot' events have raised lifesaving funds to advance care and accelerate research for MDA families living with muscular dystrophy, ALS, and related neuromuscular diseases.
By Muscular Dystrophy Association · Via GlobeNewswire · April 10, 2023
Muscular Dystrophy Association Announces Recipient of 2023 MDA Legacy Award for Achievement in Clinical Research is Merit Cudkowicz, MD, MSC, Renowned Neurologist and Clinical Researcher in ALS
New York, NY, March 06, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today announced Merit Cudkowicz, MD, MSC, a renowned neurologist and clinical researcher in ALS (amyotrophic lateral sclerosis, aka Lou Gehrig’s disease, or motor neuron disease) from Massachusetts General Hospital, will receive the 2023 MDA Legacy Award for Achievement in Clinical Research. The award will be presented at the 2023 MDA Clinical & Scientific Conference on Monday, March 20, by Stanley H. Appel, MD, longtime mentor of Dr. Cudkowicz, and MDA’s ALS Care Center Director at Houston Methodist Neurological Institute. The MDA Legacy Award for Achievement in Clinical Research is an annual recognition for outstanding accomplishments in neuromuscular disease research. The presentation will take place in-person in Dallas, Texas and via live stream. Registration is open for both in-person and virtual registration here. View the full agenda here.
Muscular Dystrophy Association Announces Programming for ALS Awareness Month
MDA Virtual Learning Series on ALS features a 5-week educational program launching May 4; MDA Advocacy Institute features discussion on legislation to improve care and funding for ALS community and key programs on May 24; MDA’s Quest Podcast features ALS medical experts and community members; and critical fundraising partnerships continue with International Association of Fire Fighters, Dutch Bros and more.
By Muscular Dystrophy Association · Via GlobeNewswire · April 24, 2023
Muscular Dystrophy Association Celebrates FDA Approval of Skyclarys, First Ever Treatment for Friedreich’s Ataxia from Reata Pharmaceuticals
New York, NY, Feb. 28, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of Omaveloxolone (Skyclarys), the first ever treatment for Friedreich’s Ataxia (FA), a rare neuromuscular disease on Rare Disease Day today, February 28. Skyclarys will be made available in the United States (US) and marketed by Reata Pharmaceuticals.
By Muscular Dystrophy Association · Via GlobeNewswire · February 28, 2023
Muscular Dystrophy Association Announces Nearly $400K in Research Grants to Address Challenges in Duchenne Muscular Dystrophy Gene Therapy
New York, NY, March 13, 2023 (GLOBE NEWSWIRE) -- Today, Muscular Dystrophy Association (MDA) announced two research grants designed to address potential transgene-triggered safety concerns in Duchenne muscular dystrophy (DMD) gene therapies. Jeffrey Chamberlain, Ph.D., Neurology, Biochemistry, Medicine/Medical Genetics, McCaw Chair in Muscular Dystrophy and Professor at the Institute for Stem Cell & Regenerative Medicine at the University of Washington, receives a $200,000 grant for a two-year study on expression of enhanced dystrophins via Adeno-associated viruses (AAV) to circumvent immune responses linked to a subpopulation of DMD patients. Carrie Miceli, Ph.D., Professor of Microbiology, Immunology and Molecular Genetics, Co-Director, Center for Duchenne Muscular Dystrophy, UCLA School of Medicine, receives $199,962 for a two-year study of single cell transcriptomics to assess transgene related molecular responses in DMD. These awards arise as a result of the MDA Gene Therapy Summit in 2022 in which global leaders from the field of gene therapy convened to discuss safety issues and challenges in the clinical translation of AAV-based gene therapies. A meeting report summarizing the presentations is available pre-press in the Journal of Neuromuscular Diseases here.
By Muscular Dystrophy Association · Via GlobeNewswire · March 13, 2023
Muscular Dystrophy Association Announces Five Recipient Organizations of Advocacy Collaboration Grants
New York, NY, Feb. 16, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today announced the five organizations receiving $109,065 in grant funding. The grants will promote and support MDA’s key public policy and advocacy initiatives in coordination with awardees to empower people living with neuromuscular diseases to live longer, more independent lives. In the spirit of MDA's commitment to collaboration, the organization is utilizing this grant program to facilitate impactful and cooperative advocacy projects and initiatives. Grantees in this innovative model are: All Wheels Up, Cure CMD, Cure Rare Disease, OPMD Association, Tufts University Medical Center (Dr. James Chambers and team), for the following projects:
By Muscular Dystrophy Association · Via GlobeNewswire · February 16, 2023
Muscular Dystrophy Association and Buffalo Bills Running Back Nyheim Hines Launch Nation's Largest St. Patrick’s Day Fundraising Campaign
Hines continues his role as the 2023 MDA National Spokesperson and joins thousands of retail locations nationwide to support MDA Shamrocks fundraising campaign throughout February and March.
By Muscular Dystrophy Association · Via GlobeNewswire · February 15, 2023
Muscular Dystrophy Association’s Venture Philanthropy Program Announces Investment in Non-viral Gene Therapy
New York, NY, Jan. 25, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) announces a $650,000 investment in Myosana Therapeutics via its MDA Venture Philanthropy (MVP) program. Myosana is a start-up company co-founded in 2018 from the University of Washington based on a non-viral antibody-mediated platform technology developed for the delivery of full-length genes to skeletal and cardiac muscles.
By Muscular Dystrophy Association · Via GlobeNewswire · January 25, 2023
Muscular Dystrophy Association Announces 2023 National Ambassador Leah Zelaya from New York
New York, NY, Jan. 23, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) has  selected 15-year-old Leah Zelaya from Brooklyn, New York, as its 2023 MDA National Ambassador. 
By Muscular Dystrophy Association · Via GlobeNewswire · January 23, 2023
Registration Opens for MDA Summer Camp for Children with Neuromuscular Diseases
Families and volunteers can elect either in-person or virtual options for the best week of the year!
By Muscular Dystrophy Association · Via GlobeNewswire · January 17, 2023
FDA’s Peter Marks, M.D., Ph.D. to deliver Keynote Address at 2023 Muscular Dystrophy Association Clinical & Scientific Conference
New York, NY, Jan. 10, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today announced Peter Marks, M.D., Ph.D. as the Keynote Speaker at the MDA 2023 Clinical & Scientific Conference. His presentation will take place Monday, March 20, 2023, in-person in Dallas, TX, and via live stream. Registration for the global convening of the worldwide neuromuscular disease community is open for both in-person and virtual registration here.
By Muscular Dystrophy Association · Via GlobeNewswire · January 10, 2023
Muscular Dystrophy Association Elects Governor Brad Henry as Chairman and Christopher Rosa, Ph.D. as Vice Chairman of the Board of Directors
MDA’s Board of Directors comprises experts from across the country with longstanding commitment and connection to the mission for the neuromuscular disease community.
By Muscular Dystrophy Association · Via GlobeNewswire · January 9, 2023