Author Archives: Joana Carvalho

Deleting Mss51 Gene Reduces Fatigue, Increases Endurance in DMD Mice

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…

Top 10 Muscular Dystrophy Stories of 2020

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…

Sarepta Poised to Start Next Trial of SRP-9001 for Duchenne MD

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…

Patient Feedback Being Sought on Puldysa for Possible NHS Funding

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…