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Research Updates

Wave Life Sciences Announces Positive Phase 1 Results for WVE-210201 in Duchenne Muscular Dystrophy (DMD)

WVE-210201 safety and tolerability profile supports Phase 2/3 clinical trial initiation in boys with DMD amenable to exon 51 skipping Delivery of interim efficacy data from ongoing open-label extension study expected in H2 2019 CAMBRIDGE, Mass., Dec. 06, 2018 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (NASDAQ:WVE), a biotechnology company focused on delivering transformational therapies for […]


Taiho’s TAS-205 May Represent New Therapy Option for DMD, Phase 1 Data Shows

Early safety and efficacy data from a Phase 1 trial suggest that Taiho Pharmaceutical’s investigational compound TAS-205 may hold the potential to treat patients with Duchenne’s muscular dystrophy (DMD). The results were reported in the study, “A phase I study of TAS-205 in patients with Duchenne muscular dystrophy,” published in Annals of Clinical and Translational Neurology. DMD is a […]


Catabasis Pharmaceuticals Announces Publication of Phase 1 Clinical Results of Edasalonexent (CAT-1004) in Duchenne Muscular Dystrophy

— MoveDMD Trial Data Showed Edasalonexent Was Well-Tolerated with No Safety Signals and Confirmed NF-kB Target Engagement — CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that data from the Phase 1 MoveDMD clinical trial of edasalonexent were published in the Journal of Neuromuscular Diseases, “Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-kB […]


Capricor to Meet with FDA under its RMAT Designation to Discuss HOPE-2 Clinical Trial

Focus will be on Milestones for Development and Commercialization CAP-1002 Therapy for Duchenne Muscular Dystrophy LOS ANGELES, Nov. 12, 2018 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ: CAPR) announced today that it will meet with the U.S. Food and Drug Administration (FDA) in December to discuss clinical trial design, surrogate or intermediate endpoints and manufacturing processes for CAP-1002, […]


Capricor’s Vesicle-based Therapy Holds Promise to Treat DMD, Preclinical Data Suggests

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 […]


GsMTx4 a Promising ‘Out-of-the-Box’ Therapy for Advanced DMD, Mouse Study Shows

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

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 […]


Sarepta Gene Therapy Appears To Help Fourth Boy With Duchenne Muscular Dystrophy

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 […]


Catabasis Pharmaceuticals Initiates Phase 3 PolarisDMD Clinical Trial For Edasalonexent In Duchenne Muscular Dystrophy

– Pivotal Study of Novel Inhibitor of NF-kB, a Key Driver of Skeletal Muscle Disease and Cardiomyopathy in Duchenne — CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the initiation of PolarisDMD, the Company’s Phase 3 trial for edasalonexent in Duchenne muscular dystrophy (DMD). Edasalonexent inhibits NF-kB, which is the key link […]


Sarepta’s Duchenne MD trial back on schedule

Sarepta’s early-stage clinical trial for a gene therapy for Duchenne muscular dystrophy is back on schedule, the Cambridge biotech said Monday. The Food and Drug Administration had put the trial on hold in July. The FDA ordered the hold after small fragments of DNA turned up in the treatment, which is being used at Nationwide […]