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Myotonic dystrophy type 1 (DM1) patients in a Phase 1/2 clinical trial saw gains in motor function and cognitive measures after receiving Dyne Therapeutics’ zeleciment basivarsen (z-basivarsen), previously known as DYNE-101, supporting the recent initiation of a Phase 3 study. Findings from the Phase 1/2 ACHIEVE trial (NCT05481879) and the design of the HARMONIA Phase…

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Sarepta Therapeutics, Inc. has announced screening and enrollment are underway in Cohort 8 of the company’s ENDEAVOR study. ENDEAVOR is an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS in multiple cohorts of individuals living with Duchenne, and Cohort 8 aims to assess prophylactic sirolimus treatment as part of an enhanced safety…

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Treatment with Duvyzat (givinostat) was associated with functional gains in boys with Duchenne muscular dystrophy (DMD) regardless of the final dose they received, according to new analyses from a Phase 3 clinical trial. The treatment also led to a slower loss of functional muscle tissue and a slower replacement of muscle by fat on MRI analyses.…

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Sarepta therapeutics is seeking to enroll 25 non-ambulant individuals with Duchenne in a clinical trial evaluating the use of sirolimus as part of an enhanced immunosuppressive regimen prior to and after Elevidys treatment.  This data will aim to determine whether sirolimus treatment can help reduce the risk of acute liver injury with AAV gene therapy. …

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Richard Farrell Jr. is 19 years old and lives in Pennsylvania. He was diagnosed with Becker muscular dystrophy (BMD) when he was 5 years old. He loves to repair computers, play musical instruments, play video games, and talk with his friends. Richard “Richie” Farrell Jr. Living with a disability has required me and my parents…

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SGT-003, an investigational gene therapy for Duchenne muscular dystrophy (DMD), worked as expected to increase levels of microdystrophin — a version of the muscle-protecting protein that’s deficient in DMD — and preserve muscle health for boys in a clinical trial, according to new data. The one-time treatment was also well tolerated, avoiding the signs of liver…

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I’m one of those people who can get lost deep in thought. As a busy mom to many in a bustling household that could seem like chaos to others, my thoughts are often my only quiet time. I share seven children with my husband, Jason: Lexi, 24; Max, 20; Chance, 18; Rowen, 17; Charlie, 15;…

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Each year, the Duchenne and Becker muscular dystrophy community comes together in Washington, DC with a shared mission: to ensure that the voices of individuals living with Duchenne and Becker and their families are heard where policy decisions are made. This year, PPMD’s Advocacy Conference once again demonstrated the extraordinary power of community advocacy. Families,…

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PGN-EDODM1, Pepgen’s experimental therapy for myotonic dystrophy type 1 (DM1), was generally well tolerated in an early clinical trial, with biomarker data suggesting that the treatment is affecting its intended molecular target.  Based on these early findings, Pepgen is now sponsoring a Phase 2 trial, FREEDOM2-DM1 (NCT06667453), to evaluate the safety of repeated doses of…

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Capricor Therapeutics has announced additional analyses and new functional outcomes data from the Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne. Deramiocel is the company’s investigational cell therapy for the treatment of Duchenne cardiomyopathy. Cardiac MRI analyses demonstrated Deramiocel’s impact on the heart, with data showing reduction in fibrotic segments in patients treated with…

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