Category Archives: Muscular Dystrophy Treatment

WATCH TIME: 9 minutes "Our job today is far from done in this disease, and we've improved the whole therapeutic landscape of exon skipping. Continue reading →

Oral treatment withEDG-5506, now known as sevasemten, continued to stabilize muscle function after two years in men with Becker muscular dystrophy(BMD) taking part in a Phase 1 trial. Thats in contrast to the progressive deterioration of motor function seen during the natural course of BMD in the absence of treatment. Continue reading →

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Duchenne muscular dystrophy (DMD) is a debilitating condition that predominantly affects males, causing progressive muscle degeneration and weakness. Continue reading →

What is Muscular Dystrophy? Muscular dystrophy comprises a group of conditions causing progressive muscle weakness and reduced flexibility Continue reading →

On Jan. 8, 2020, as I was parking my car, I got a long-awaited phone call from one of my sons doctors Continue reading →

WATCH TIME: 3 minutes "One possibility is that prescribing physicians arent aware of the age range that received the drug in its pivotal trials or have knowledge on the FDA-approval process. When patients receive the drug at a later stage of their DMD, it might impact the predicted effectiveness of the medications." For years, Duchenne muscular dystrophy (DMD) was mainly treated with a combination of corticosteroids, assistive devices, and cardiac and respiratory support until 2016, when the exon-skipping therapies began to emerge. The first approved therapy was eteplirsen (Exondys 51; Sarepta Therapeutics), an antisense oligonucleotide designed to increase dystrophin, followed by other agents, which include golodirsen, viltolarsen, and casimersen. Continue reading →

By: Josh Baxt | January 23, 2024 | 7 min. read| Share Article Summary Amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), and spinal muscular atrophy (SMA) are devastating disorders that affect nerve and muscle function. Until recently, they have been difficult, if not impossible, to treat. Continue reading →

Sarepta Therapeutics has started screening participants for a Phase 3 clinical trial called EMERGENE that will test its gene therapy candidate SRP-9003in children withlimb-girdle muscular dystrophy type 2E(LGMD2E). The open-label trial, also known as SRP-9003-301, aims to recruit 15 patients, age 4 and older, with or without the ability to walk independently. Continue reading →

New York, Jan. Continue reading →