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Early trial data support RNA therapies for two muscular dystrophy types
SRP-1001 and SRP-1003, Sarepta Therapeutics’ investigational RNA-based therapies for two types of muscular dystrophy, were generally well tolerated in early results, according to data from two Phase 1/2 trials. SRP-1001, for facioscapulohumeral muscular dystrophy type 1 (FSHD1), and SRP-1003, for myotonic dystrophy type 1, showed high levels of delivery to muscle in early company-reported data.…
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Quest Podcast: Pizazz, Purpose, and Periodic Paralysis: How Cienna Ditri Turns Lived Experience into Advocacy
In this Quest Podcast episode, we chat with advocate, social media influencer, and President of the Periodic Paralysis Association (PPA), Cienna Ditri, who lives with periodic paralysis. Cienna shares her diagnostic journey — from childhood soccer games where something felt “off” to finally getting answers — and how living with an unpredictable condition has shaped…
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Figuring out where to buy a lift chair for my FSHD symptoms was no small task
Recently, standing up has become even more difficult for me than it previously was. The last few times I sat in my comfy recliner in my music and writing room, I worried I might have to call 911 for help getting up. My son, Ryan, had already added 3-inch risers to the bottom of the…
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Wildland Fire Fighter Refuses to Allow Diagnosis to Define His Life
When Tyler Long was diagnosed with facioscapulohumeral muscular dystrophy (FSHD) during his senior year of high school, doctors advised him that this diagnosis meant that he should plan his future around a “non-strenuous” lifestyle. For the active and athletic outdoorsman, this suggestion wasn’t in alignment with how he saw himself and how he wanted to…
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Sarepta plans to seek full FDA approval of 2 Duchenne treatments
Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant full approval for Amondys 45 (casimersen) and Vyondys 53 (golodirsen), two exon-skipping therapies designed to treat certain people with Duchenne muscular dystrophy (DMD). The company said it met with the FDA to discuss plans for the two therapies, and the FDA…
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PPMD Provides $250,000 in Funding to Secretome Therapeutics Through PPMD Venture Pathways Program to Support Development of Cardiac Cell Therapy
PPMD and Secretome Therapeutics (Secretome) are excited to announce that PPMD has provided $250,000 in funding to Secretome through PPMD Venture Pathways, our venture-philanthropy initiative that provides funding to industry to accelerate therapeutic development for Duchenne and Becker. This funding commitment aims to advance the development of STM-01, Secretome’s neonatal cardiac progenitor cell (nCPC) product…
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When it comes to rare disease advocacy, just keep showing up
According to the EveryLife Foundation for Rare Diseases, more than 30 million Americans live with at least one rare disease. Considering that’s nearly 10% of the U.S. population, these diseases don’t seem so rare after all. For decades, the disability rights movement has fought for the rights of this population, advocating for greater accessibility and…
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MDA 2026: Dyne advances DMD therapy z-rostudirsen toward approval
Dyne Therapeutics is advancing its investigational exon-skipping therapy zeleciment rostudirsen (z-rostudirsen), formerly known as DYNE-251, toward regulatory approval after trial data showed early signs of benefit across multiple disease measures in boys with Duchenne muscular dystrophy (DMD). The most recent data from the Phase 1/2 DELIVER clinical trial (NCT05524883) showed that the therapy increased levels of…
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MDA 2026: This year’s MDA meeting provided a glimpse into the future
Muscular Dystrophy News Today is wrapping up coverage of the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which ran from March 8-11 in Orlando, Florida, and online. This year, more than 2,400 attendees from 40 countries gathered in person and virtually to discuss recent advances in neuromuscular disease research and care — and…
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How to Make Vocational Training Affordable
There are many reasons a person with a disability might choose vocational training. One prominent reason: It can provide an affordable, direct pathway to employment, making it an attractive option for anyone interested in entering the workforce. Vocational training, also called trade school or technical education, provides training targeted to a specific career. Unlike traditional…
