A potential exon-skipping therapy for Duchenne muscular dystrophy (DMD) called DS-5141 showed a good safety profile with repeat dosing in a small clinical trial, according to a press release. The Phase 1/2 clinical trial (NCT02667483) tested DS-5141 for a first time in Duchenne patients amenable to exon 45 skipping. No…
The first boy has been dosed in a Phase 3 trial testing Pfizer‘s gene therapy, PF-06939926, in treating Duchenne muscular dystrophy (DMD). “The initiation of our pivotal trial, which is the first Phase 3 DMD gene therapy program to begin enrolling eligible participants, is an important milestone for the…
The investigational gene therapy SRP-9001 safely leads to the production of a functional form of dystrophin in boys with Duchenne muscular dystrophy (DMD) — and to improvements in their motor abilities, according to top-line results of a Phase 2 trial. However, following almost one year of the Sarepta…
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…
AMO Pharma has announced the initiation of REACH-CDM, a pivotal Phase 2/3 clinical trial that will assess the efficacy and safety of the investigational medication AMO-02Â (tideglusib) in children and adolescents with congenital myotonic dystrophy type 1Â (CDM1), also known as Steinert disease. The double-blind trial (NCT03692312) is…
Throughout 2020, Muscular Dystrophy News Today brought you daily coverage of important discoveries, treatment advancements, clinical trial findings, and other relevant events related to muscular dystrophy. As we look forward to bringing you more news this year, we present here the 10 most-read stories of 2020, along with a…
Edgewise Therapeutics has received $95 million in funding to advance the clinical development of its experimental therapy EDG-5506, designed to improve physical functioning in people with Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD).
The French National Agency for Medicines and Health Products Safety has cleared Généthon to begin a clinical trial testing the safety and efficacy of GNT0004, a potential gene therapy for Duchenne muscular dystrophy (DMD), in France. “From translational research to clinical application, Généthon has supported this candidate drug,…
Sarepta Therapeutics’ investigational treatment SRP-5051 enhanced cell penetration, exon skipping, and dystrophin levels at a lower dose than its Exondys 51 (eteplirsen) therapy in a Phase 2 clinical trial in patients with Duchenne muscular dystrophy (DMD).
Translarna (ataluren) has been approved in Russia for treating Duchenne muscular dystrophy (DMD) caused by nonsense mutations in patients ages 2 and older. “Translarna was the first therapeutic ever approved for nonsense mutation Duchenne muscular dystrophy patients and we are excited to expand approval of Translarna into Russia,” Stuart…
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