Patricia Inácio, PhD,  science writer—

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…

Using the results of a New York State pilot testing program as evidence, Parent Project Muscular Dystrophy (PPMD) submitted its proposal seeking the addition of Duchenne muscular dystrophy (DMD) to the nationwide newborn screening panel. The proposal nominates DMD for inclusion in the Recommended Uniform Screening Panel or RUSP…

FibroGen has completed patient enrollment for its Phase 3 trial evaluating pamrevlumab in combination with corticosteroids as a potential treatment for children with Duchenne muscular dystrophy (DMD). The LELANTOS-2 study (NCT04632940) has enrolled 73 boys with DMD, ages 6 to 12, who maintain their ability to walk.

New data from a Phase 1/2 clinical trial of an exon-skipping gene therapy suggest it may help preserve muscle function if delivered early on to Duchenne muscular dystrophy (DMD) patients with exon duplications. This is the first time a clinical trial has shown production of full-length dystrophin, the protein…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to elamipretide for treating Duchenne muscular dystrophy (DMD). According to Stealth BioTherapeutics, the therapy’s developer, elamipretide has been found to help improve the function of mitochondria — the powerhouses of cells — that…

Santhera Pharmaceuticals and ReveraGen BioPharma will seek approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD) in the U.S. this year, with a filing expected to be completed by the end of June. The submission of the companies’ rolling new drug application (NDA) to the U.S. Food and Drug Administration…

Fulcrum Therapeutics plans to launch a Phase 3 trial of losmapimod, a potential oral treatment for facioscapulohumeral muscular dystrophy (FSHD), by June. The announcement of the trial, called REACH, follows clinically relevant benefits seen in the Phase 2b ReDUX4 trial (NCT04003974) and consultations with key regulators, including…