Jesse's Journey Foundation

Capricor Therapeutics’ vesicle-based therapy CAP-2003, derived from the company’s proprietary cardiosphere-derived cells (CDCs), may be an effective therapy for Duchenne muscular dystrophy (DMD), according to preclinical data. The company presented the results in August in two scientific posters at the 2018 Gordon Research Conference on Extracellular Vesicles in Newry, Maine. Capricor’s lead candidate for the treatment of DMD is CAP-1002, which is […]

A new investigational therapy called GsMTx4, developed by researchers at the University of Buffalo and based on a molecule found in tarantula venom, is able to prevent the loss of muscle mass and muscle injury in a mouse model of advanced Duchenne muscular dystrophy (DMD). These findings were published in the study, “GsMTx4-D provides protection to the D2.mdx mouse,” […]

PTC Therapeutics Announces Initial Data from Patient Registry Demonstrating Translarna™ (ataluren) Slows Disease Progression in Children with Duchenne Caused by a Nonsense Mutation SOUTH PLAINFIELD, N.J., Oct. 6, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced preliminary data from the first international drug registry for Duchenne patients receiving Translarna™ (ataluren), underscoring the long-term clinical benefit of Translarna when […]

An experimental gene therapy dramatically improved the ability of a young boy with Duchenne muscular dystrophy to rise from the ground and climb stairs, repeating and improving on results seen in three previous patients who received the treatment, which is being developed by Sarepta Therapeutics of Cambridge, Massachusetts. “The cautionary note is this all has […]