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Research Updates

Wave Life Sciences Duchenne Muscular Dystrophy Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilot Program

CAMBRIDGE, Mass., Jan. 03, 2019 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (NASDAQ:WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the planned Phase 2/3 efficacy and safety trial for its lead Duchenne muscular dystrophy (DMD) clinical program has been selected for the U.S. Food and […]


Project Hercules Enlists Industries Help to Make Duchenne Therapies Affordable

Emily Crossley was a well-known TV correspondent for Britain’s Channel 4, and a presenter for CNN International’s World Business Today program. But her own world was turned upside-down the day she learned her son, Eli, had Duchenne muscular dystrophy (DMD). “The initial diagnosis was so devastating. There’s no escape. It’s absolutely relentless,” she said. “When […]


Catabasis Pharmaceuticals Phase 3 PolarisDMD Clinical Trial for Edasalonexent in Duchenne Muscular Dystrophy Progress Update and Additional Trial Sites Open for Enrollment

— Patient Enrollment Underway in U.S. with Nine Locations Open for Enrollment — — Global Start-Up Activities Ongoing with Clinical Trial Application Approvals Received in Canada and Multiple European Countries — CAMBRIDGE, Mass., January 3, 2019 – Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today shared an update on clinical trial sites in the United […]


Potential DMD Therapy WVE-210201 Shows Positive Phase 1 Safety Results

WVE-210201, an exon 51 skipping therapy, has shown positive safety and tolerability results in a Phase 1 clinical trial in boys with Duchenne muscular dystrophy (DMD), Wave Life Sciences announced. According to Wave Life Sciences, the positive safety data supports moving the therapy into a Phase 2/3 trial, planned to start in 2019, to confirm the safety profile and establish efficacy. The company […]


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, […]