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Solid Biosciences announced a widely anticipated update on their INSPIRE DUCHENNE Phase 1/2 clinical trial of SGT-003
Solid Biosciences announced a widely anticipated update on their INSPIRE DUCHENNE Phase 1/2 clinical trial of SGT-003, their investigational micro-dystrophin gene therapy for Duchenne muscular dystrophy. 15 participants have received SGT-003 so far across sites in the U.S., Canada, Italy, and the UK. No serious treatment-related adverse effects have been reported, and early safety results…
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Hidden role of MYOD protein may lead to new MD therapies: Study
A protein called MYOD, which is known to control the growth of muscle stem cells during muscle repair, has long been known to be able to turn on genes, but a recent study has now found that MYOD can also turn off certain genes. According to researchers, the findings may help pave the way toward…
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On the importance of human connection
There is power in connection. As humans, we all learn this truth at a young age, starting with the people around us when we’re little: our family. As we grow, we connect with other kids at school, in church, and elsewhere in the community, and just by existing every day. We begin to understand that…
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Update to the U.S. Duchenne Community on the Ataluren (Translarna) for Nonsense Mutation Duchenne Muscular Dystrophy
The FDA is continuing its review of Ataluren (Translarna), the first potential therapy specifically for individuals with nonsense mutation Duchenne (nmDMD). There is currently no set decision date. The application includes more than 10 years of safety and efficacy data. Read the letter to the community below: PTC Update to the Duchenne Community Aug 2025Download…
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In Case You Missed It…
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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Entrada Therapeutics doses first participant in Exon 44 skipping program; provides updates to other exon-skipping programs
Entrada Therapeutics, which received early funding from CureDuchenne, has provided updates on the status of several exon skipping programs. We are pleased to share that they have dosed the first participant in the ELEVATE-44-201 clinical trial for individuals with Duchenne amenable to skipping Exon 44. They expect to report clinical data from the first cohort…
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Disabled artists’ challenges can propel creative solutions
As an artist with Duchenne muscular dystrophy (DMD), I’ve long recognized how creativity can foster meaningful connection, perhaps especially when rooted in design that’s accessible and inclusive. My partner, Amanda, and I recently spoke at GovTech’s Inclusive Design Week 2025 here in Singapore as part of the closing panel discussion, “Impact by Design.” GovTech is…
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FSHD patients sought for first clinical trial testing EPI-321
Enrollment is now ongoing in a first-in-human trial testing EPI-321, an epigenetic therapy designed to address the root cause of facioscapulohumeral muscular dystrophy (FSHD), a disease type in which symptoms like muscle weakness typically become apparent by adulthood. The Phase 1/2 clinical trial (NCT06907875) is expected to enroll nine people with FSHD, ages 18-75, all…
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Behind the Drug: Nusinersen (Spinraza) for SMA
Spinal muscular atrophy (SMA) is a rare genetic disease that affects the peripheral nervous system and voluntary muscle movement. It’s estimated that it affects 1 in 11,000 live births in the United States. People with SMA lose motor function over time, but the rate and extent of loss depend on the type of SMA. SMA…
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DYNE-251 for DMD granted FDA breakthrough therapy designation
The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to Dyne Therapeutics’ DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. This designation is intended to accelerate the development and regulatory review of medications intended to treat serious conditions. Benefits include enhanced FDA support in…
