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In total, 18 have been published by CureDuchenne which Ground News has aggregated in the past 3 months.

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Top CureDuchenne News

REGENXBIO AFFINITY DUCHENNE: Trial of Investigational RGX-202 Gene Therapy

In this webinar, Dr. Naz Dastgir, Executive Medical Director and Clinical Development Lead for RGX-202 at REGENXBIO, shared information on the active and enrolling AFFINITY DUCHENNE trial and how families can obtain additional information about RGX-202 and potential clinical trial participation.Watch HEREThe post REGENXBIO AFFINITY DUCHENNE: Trial of Investigational RGX-202 Gene Therapy appeared first on CureDuchenne.Read Article

United States · United States

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 remain positive. The company plans to dose a minimum of 20…Read Article

FDA · United States

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 development and decision-making, regular communication wi…See the Story

Update to the U.S. Duchenne Community on the Ataluren (Translarna) for Nonsense Mutation Duchenne Muscular Dystrophy

Nonsense Mutation Duchenne Muscular DystrophyThe 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 2025DownloadThe post Update to the U.S. Duchenne Commu…United States · United States Read Article

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 during the first half of 2026. This study is currently enrolling ambulatory particip…United Kingdom · United Kingdom Read Article

Brogidirsen and the Promise of Exon 44 Skipping: New Horizons in RNA Therapy for Duchenne Muscular Dystrophy – Patient Worthy

Duchenne muscular dystrophy (DMD) is a devastating, inherited muscle-wasting disorder caused by mutations in the DMD gene, leading to an absence of functional dystrophin protein. While current standards of care, such as corticosteroids and supportive therapies, have extended patient lifespans and improved quality of life, they remain largely palliative and do not address the root […]See the Story

Sarepta : Restructuring and Elenidys - Strategic Plan

Sarepta: Restructuring and Elenidys – Strategic Plan Sarepta Therapeutics: Restructuring Massive after Deborations in Genic Therapy – Urgent News The pharmaceutical company Sarepta... The post Sarepta: Restructuring and Elenidys – Strategic Plan appeared first on World News.United States · United States See the Story

Why Is Capricor Therapeutics Stock Trading Lower On Friday? - Capricor Therapeutics (NASDAQ:CAPR)

67% Center coverage: 6 sources

Capricor Therapeutics Inc. (NASDAQ:CAPR) stock is trading lower on Friday. The company received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for Deramiocel, the company’s lead cell therapy candidate for cardiomyopathy associated with Duchenne muscular dystrophy (DMD). In the CRL, the FDA stated that it had completed its application review but could not approve…FDA · United States See the Story

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