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CureDuchenne provided early funding to Entrada Therapeutics.  Entrada has received regulatory authorization in Europe to initiate a Phase 1/2 clinical trial of ENTR-601-44 in ambulatory individuals with Duchenne amenable to skipping exon 44.  The study, which will run in the UK and the EU, will start in the 2nd quarter of 2025.  Entrada is also…

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Parent Project Muscular Dystrophy (PPMD), a nonprofit dedicated to ending Duchenne muscular dystrophy (DMD), has published an updated edition of the PJ Nicholoff Steroid Protocol, marking the 10th anniversary of its first release. Called PJ’s Protocol, the guidance is designed to help healthcare providers manage corticosteroid use in DMD, particularly during times of physical stress…

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Ten years ago, the Duchenne community gained a vital tool for the clinical management of adrenal insufficiency and steroid stress dosing for individuals on long-term corticosteroid therapies: the PJ Nicholoff Steroid Protocol for Healthcare Providers. What began as a response to a deeply personal experience has become an essential resource for families and clinicians and…

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PPMD is closely monitoring the U.S. House of Representatives Energy and Commerce Committee markup of the Committee’s budget reconciliation text, scheduled for tomorrow, Tuesday, May 13, 2025, at 2:00 PM ET. Updated legislative language released yesterday includes proposals that would modify certain Medicaid policies, including those related to work requirements and provider taxes. According to…

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Jaylin Hsu, from San Marino, California, is senior at the University of California, Los Angeles studying Molecular, Cell, & Developmental Biology. Since his freshman year, he has been involved in neuromuscular disease clinical research and plans to attend medical school. His passion stems from his scientific work and interactions with countless patients diagnosed with muscular…

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At the Muscular Dystrophy Association (MDA), we’re marking a major moment in our history. Dr. Donald S. Wood, who has led MDA as President and CEO since 2020, has announced his decision to retire. Dr. Don Wood This transition brings both celebration and reflection. For nearly five decades, Dr. Wood has been part of MDA’s…

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Solve FSH, a venture philanthropic organization, has invested $3 million to support the development of Armatus Bio‘s microRNA-based therapy ARM-201 as a treatment for facioscapulohumeral muscular dystrophy (FSHD). ARM-201 is designed to block the production of DUX4, the protein that is abnormally produced in people with FSHD, a type of muscular dystrophy that primarily affects the muscles…

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On April 3, 2025, the U.S. Department of Health and Human Services (HHS) announced the dissolution of the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), an unexpected development that has left a critical gap in the federal process for reviewing and recommending conditions for the Recommended Uniform Screening Panel (RUSP). For the…

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Monty Nye The mission to find treatment for amyotrophic lateral sclerosis (ALS) hits especially close to home for fire fighters, with research revealing that they are twice as likely to develop the disease. For Monty Nye, his commitment to the fight to end ALS is fueled by a deeply personal connection – and provides a…

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Quest Media is an innovative adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities. With so many valuable…

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