Patricia Inácio, PhD, science writer —

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…

Muscular Dystrophy News Today brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to muscular dystrophy (MD) throughout 2021. We look forward to reporting more news to patients, family members, and caregivers dealing with MD during 2022. Here are the top 10 most-read articles of…

A branch of the U.S. Food and Drug Administration (FDA) known as the Division of Cardiology and Nephrology has given positive feedback to Stealth BioTherapeutics‘ program for Duchenne muscular dystrophy (DMD). In a pre-investigational new drug (pre-IND) meeting, the division — called the DCN — agreed to the design of…

A Phase 1 trial of SRP-9001, Sarepta Therapeutics‘ investigational gene therapy, is recruiting 3-year-old boys with Duchenne muscular dystrophy (DMD) who are able to walk for a new patient group in California, according to a press release from the Muscular Dystrophy Association. This group, being enrolled at the…

Tamoxifen, commonly used to treat breast cancer, failed to meet its primary goal — delaying disease progression — in a Phase 3 study evaluating its use in boys with Duchenne muscular dystrophy (DMD), preliminary data show. Boys treated with tamoxifen and those given a placebo over 48 weeks in the…

CureDuchenne has teamed up with Brigham and Women’s Hospital, in Boston, to launch the nation’s first free newborn screening initiative for Duchenne muscular dystrophy (DMD). The partnership establishes Brigham and Women’s as the only U.S. birth hospital to offer parents the choice of screening their new baby for…