Patricia Inácio, PhD, science writer —

AOC 1020, Avidity Biosciences’ investigational therapy for facioscapulohumeral muscular dystrophy (FSHD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The designation is intended to encourage the development of therapies for rare diseases, those affecting fewer than 200,000 people in the U.S. It provides…

PGN-EDODM1, a therapy candidate for myotonic dystrophy type 1 (DM1), showed a favorable safety profile in preclinical studies, including those in non-human primates, PepGen, the company developing the treatment, announced. This work is expected to support a request to initiate a single ascending dose clinical trial of PGN-EDODM1…

Sarepta Therapeutics‘ investigational GALGT2 gene therapy helped stabilize muscle function when delivered at a higher dose to a boy with Duchenne muscular dystrophy (DMD), data from a Phase 1/2a clinical trial show. The other trial participant, a boy with more severe disease, had a slight lung function improvement…

EDG-5506, Edgewise Therapeutics’ experimental oral therapy, continues to safely reduce muscle damage and improve physical function in adults with Becker muscular dystrophy (BMD), according to six-month interim data from the Phase 1 ARCH trial. Treatment led to a marked reduction in the average levels of key markers of muscle…

Dystrogen Therapeutics’ experimental cell therapy, DT-DEC01, continues to show signs of efficacy in three boys with Duchenne muscular dystrophy (DMD), who exhibited improved muscle strength and motor function after six months in a clinical trial. All patients showed signs of clinical improvements irrespective of their mutation in the…

A first patient has been dosed in Denmark as part of a Phase 1/2 trial of ATA-100, Atamyo Therapeutics’ one-time gene therapy for a specific form of limb-girdle muscular dystrophy called LGMD-2I or LGMD-R9. “This is an exciting milestone for our company but most importantly, if this clinical trial is…

Treatment with Exondys 51 (eteplirsen) outperformed standard of care therapy at delaying respiratory decline in boys with Duchenne muscular dystrophy (DMD), an analysis of data from several clinical trials found. Compared with boys on other therapies, those treated with Exondys 51 in trials had “a delay of…

A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases have been selected to receive a Horizon Therapeutics‘ #RAREis Global Advocate Grant. Winners of this year’s inaugural awards are spread across nine countries and represent a total of 29 rare…

The first patient has been dosed in a pivotal U.S. Phase 3 study evaluating the investigational cell therapy CAP-1002 in males with advanced Duchenne muscular dystrophy (DMD). The trial, called HOPE-3 (NCT05126758), will evaluate the effect of CAP-1002 in upper limb function, among other assessments, in about…

A Phase 2 clinical trial testing Edgewise Therapeutics’ investigational oral therapy EDG-5506 is recruiting boys and men with Becker muscular dystrophy (BMD). Called CANYON (NCT05291091), the study plans to enroll up to 66 BMD patients, ages 12–50, across 14 clinical sites in the U.S., U.K., and the…