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Entrada Therapeutics announced positive initial data from their Phase 1/2 ELEVATE-44-201 study in DMD patients amenable to skipping exon 44.
Entrada Therapeutics, who received early funding from CureDuchenne to advance their Endosomal Escape Vehicle Platform, today announced positive initial data from their Phase 1/2 ELEVATE-44-201 study in DMD patients amenable to skipping exon 44. Read press release HERE The post Entrada Therapeutics announced positive initial data from their Phase 1/2 ELEVATE-44-201 study in DMD patients…
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Companies join forces to speed production of 1-time treatment for FSHD
Epicrispr Biotechnologies is partnering with Forge Biologics to support the development of EPI-321, Epicrispr’s one-time epigenetic treatment candidate for facioscapulohumeral muscular dystrophy (FSHD). The treatment uses epigenetics — chemical modifications that can alter gene activity without changing a gene’s DNA sequence — to turn off the abnormally-activated gene that underlies FSHD. According to the developer,…
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The Joy (and Lessons) of Motherhood from MDA Ambassadors
Motherhood is an incredible journey, layered with joy, growth, challenges, victories, and an abundance of love. For moms living with neuromuscular disease, the role of motherhood can sometimes entail additional layers, requiring extra patience, adaptability, and resilience. But, with a beautiful harmony, those very traits are often strengthened through daily life with a disability and…
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Entrada Reports Positive Topline Results from Phase 1/2 ELEVATE-44-201 Study in Duchenne
Entrada Therapeutics has announced positive topline data from Cohort 1 of the double-blind, placebo-controlled, multiple ascending dose portion of the Phase 1/2 ELEVATE-44-201 study evaluating ENTR-601-44 in ambulatory participants ages 4–20 living with Duchenne muscular dystrophy amenable to exon 44 skipping. ENTR-601-44 is an investigational therapy combining their cell penetrating peptide with an exon skipping…
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Protected: CureDuchenne Expands Global Duchenne Care Efforts Through First Latin American Symposium in Chile
This content is password protected. To view it please enter your password below: Password: The post Protected: CureDuchenne Expands Global Duchenne Care Efforts Through First Latin American Symposium in Chile appeared first on CureDuchenne.
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Momentum Continues: Duchenne Newborn Screening Advances Across the US
Duchenne was added to the Recommended Uniform Screening Panel (RUSP) (RUSP) on December 16, 2025, and in the months since, that decision, coupled with our continued advocacy efforts, have driven meaningful action at the state level. While the RUSP provides a national recommendation, each state must follow its own process to add new conditions to…
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Solid Biosciences Doses First Participant in Phase 3 IMPACT DUCHENNE Clinical Trial Evaluating SGT-003 in Duchenne
Solid Biosciences Inc. has shared that the first participant has been dosed in IMPACT DUCHENNE, the company’s multi-country, placebo-controlled, randomized, double-blind, Phase 3 clinical trial investigating SGT-003 for the treatment of Duchenne muscular dystrophy. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin via their novel AAV capsid designed…
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First Participant Dosed in Phase 3 Trial for SGT-003 in Duchenne
Solid Biosciences has announced the first participant has been dosed in the Phase 3 IMPACT DUCHENNE trial evaluating SGT-003. The global study is now underway, with active sites in Australia and Canada, and additional locations expected to open in the UK, U.S., and beyond. Meanwhile, 46 participants have already been dosed in the ongoing Phase…
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One way to process loss is by advocating for lasting change
The death of my dear friend Timothy Chan last month still affects me deeply. A couple weeks ago, I stood at his memorial, speaking about a friend who had lived fully despite the barriers associated with a lack of awareness about Duchenne muscular dystrophy (DMD) here in Singapore. Now, I’m considering how best to carry…
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STAT: A decade ago, these drugs tore apart the FDA. Today might be some patients’ best hope.
By Jason Mast Stat ArticleDownload The post STAT: A decade ago, these drugs tore apart the FDA. Today might be some patients’ best hope. appeared first on CureDuchenne.
