Author Archives: Joana Carvalho

By deleting the Mss51 gene, scientists were able to reduce fatigue, increase endurance, and ease mitochondrial impairment in a mouse model of Duchenne muscular dystrophy (DMD). Mss51 is a gene that provides instructions to make a skeletal muscle protein specifically found in mitochondria, the small cell compartments responsible for…

CureDuchenne has launched a new initiative to prioritize the needs of the Duchenne muscular dystrophy (DMD) community during the COVID-19 vaccine rollout. The nonprofit, which supports patients with DMD, their caregivers, and loved ones, aims to act as an information conduit, educating the DMD community on COVID-19 vaccination,…

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…

Edasalonexent, a potential oral therapy for Duchenne muscular dystrophy (DMD), can prevent the development of heart disease and help to preserve bone health, according to data from two studies in mouse models of DMD. Catabasis Pharmaceuticals’ investigational treatment was also well-tolerated when given for up to…

Santhera Pharmaceuticals has decided to stop SIDEROS, its Phase 3 trial assessing the effectiveness of Puldysa (idebenone) at delaying respiratory decline in patients with Duchenne muscular dystrophy (DMD) on a stable glucocorticoid dose. An interim data analysis of the study (NCT02814019), which was fully enrolled, determined that…

A single dose of SRP-9001, Sarepta Therapeutics’ investigational gene therapy for  Duchenne muscular dystrophy (DMD), continues to be safe and to improve motor function in four boys treated two years ago, according to data from an ongoing Phase 1/2 trial. Updated results from Study-101 (…

The experimental therapy vamorolone can improve motor function significantly in boys with Duchenne muscular dystrophy (DMD), without stunting their growth and with fewer side effects compared to standard corticosteroids, according to 18-month data from a Phase 2 trial and its extension study. Findings were reported in the study, “…

Sarepta Therapeutics has completed a meeting with the Office of Tissues and Advanced Therapies (OTAT) that clarified regulatory requirements to start the company’s next clinical trial of SRP-9001, an investigational gene therapy for Duchenne muscular dystrophy (DMD). During the type C “written response only” meeting, the…

The National Institute for Health and Care Excellence (NICE) is urging patient organizations to gather feedback from those with Duchenne muscular dystrophy (DMD) who received Santhera Pharmaceuticals‘ investigational treatment Puldysa (idebenone), Duchenne UK announced in a press release. This treatment is designed to slow the…

A single administration of the gene therapy SRP-9003 at a low dose continues to lead to significant improvements in functional measures in three children with limb girdle muscular dystrophy (LGMD) type 2E, one-year data from a Phase 1/2 trial show. When administered at a high dose in three…