June 2026

Capricor Therapeutics has shared that the US FDA will hold an Advisory Committee meeting on July 29, 2026, to discuss the company’s Biological License Application (BLA) seeking approval of deramiocel, an investigational cell therapy for the treatment of Duchenne.  It will be possible to watch this meeting via live streaming, and more details will be…

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Solid Biosciences has provided an update to the community about SGT-003, their AAV-delivered microdystrophin gene therapy.  In the Phase 1/2 INSPIRE DUCHENNE clinical trial, 53 boys have been dosed, ranging from 6 months to 10 years old.  Solid has also started their Phase 3 IMPACT DUCHENNE trial, with sites currently enrolling in Australia and Canada. …

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Today, Capricor Therapeutics announced positive five-year data from the company’s ongoing HOPE-2 open label extension (OLE) study of Deramiocel, Capricor’s novel cell therapy, in individuals living with Duchenne. According to Capricor, among the nine patients who remain enrolled in the HOPE-2 OLE study, treatment with Deramiocel continued to attenuate disease progression over five years, as…

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Solid Biosciences has shared a new letter to the Duchenne community providing updates on the development of SGT-003, the company’s investigational gene therapy for Duchenne. Solid shared that the ongoing INSPIRE DUCHENNE Phase 1/2 clinical trial has dosed 53 patients ages 0 to 10 years old, including a 6-month-old child, who is the youngest known…

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PPMD’s 2026 Annual Conference is in full swing! PPMD’s 2026 Annual Conference kicked off with opening remarks from Katherine Beaverson, MD, CEO of PPMD, who welcomed attendees, introduced the dedicated PPMD team, recognized the many individuals and partners who strengthen our community, and shared how PPMD is working every day to accelerate research, improve care,…

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The Department of Justice (DOJ) has released a memorandum arguing that states are not required to provide in-home or community-based care to people with disabilities, including those with muscular dystrophy (MD). The move drew sharp condemnation from the Muscular Dystrophy Association (MDA), which stated in a press release that it “will work tirelessly to protect…

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Today, Avidity Biosciences announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of delpacibart zotadirsen (del-zota) for individuals living with Duchenne who are amenable to exon 44 skipping. The application is being reviewed through the FDA’s Accelerated Approval pathway and is supported by data…

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Exon 44 Skipping CureDuchenne was an early funder of Avidity Biosciences, which is now a Novartis company.  We are pleased to share that they have submitted a BLA to the FDA for delpacibart zotadirsen (“del-zota”) as a treatment option for individuals with Duchenne amenable to skipping exon 44.  This application was submitted through the FDA’s…

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REGENXBIO has completed dosing in the confirmatory study of RGX-202, an AAV-delivered microdystrophin gene therapy for the treatment of Duchenne.  The company is on track to initiate a Biologics License Application (BLA) under the accelerated approval pathway in Q3 2026, for a potential approval by the US FDA in the second half on 2027. Press…

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Hello! In this blog, I’m sharing accessible beauty and fashion tips I’ve collected through plenty of trial and error. Beauty and fashion are major forms of self-expression for me, and I know I’m not alone in that. People with disabilities deserve to present themselves to the world in whatever way feels authentic and comfortable to…

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