Jesse's Journey Foundation

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Muscular Dystrophy Association (MDA): 2021 Conference Update

Jesse’s Journey is pleased to share three treatment updates for Duchenne muscular dystrophy as presented at the recent Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference.

Pfizer: PF-06939926

The ongoing Phase 1b study (NCT03362502) is evaluating the safety and tolerability of PF-06939926 in boys who can walk at least 10 meters (about 33 feet) without assistance.

Click the button below for the latest update (as posted on Muscular Dystrophy News).

 Solid Biosciences: SGT-001

SGT-001 is designed to deliver a gene that carries the instructions for the protein microdystrophin — a shorter but functional version of dystrophin which missing in Duchenne patients. The delivery carrier is a modified and harmless adeno-associated virus (AAV) that specifically targets muscle cells.

Click the button below for the latest update (as posted on Muscular Dystrophy News).

Santhera Pharmaceuticals and ReveraGen BioPharma: Vamorolone

Long-term treatment with the experimental steroid therapy vamorolone safely and effectively delays motor function decline in boys with Duchenne muscular dystrophy (DMD), according to final, 2.5-year data from a Phase 2 clinical trial and its extension study.

Click the button below for the latest update (as posted on Muscular Dystrophy News).

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