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Research Updates

PTC Therapeutics Announces Publication of Data Demonstrating the Clinical Differentiated Benefit of Deflazacort

– Post hoc analysis of the placebo arm of ACT DMD Data Published in Muscle & Nerve – SOUTH PLAINFIELD, N.J., July 24, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the publication of data in Muscle & Nerve comparing the efficacy and safety of deflazacort and prednisone/prednisolone from the placebo arm of the ACT DMD study.  The results […]


PTC Therapeutics Announces Positive Data from its Translarna™ Phase II Clinical Trial in Children as Young as Two Years with Nonsense Mutation Duchenne Muscular Dystrophy

SOUTH PLAINFIELD, N.J., July 9, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced the presentation of data from the Translarna (ataluren) Phase II Study 030 demonstrating that the safety and pharmacokinetic profile of Translarna in children from two to five years with nonsense mutation Duchenne muscular dystrophy (nmDMD) was consistent with that for older children.1  Importantly, the […]


Potential DMD Therapy Now in Phase 1 Trial, WVE-210201, Likely To Be Named Orphan Drug in EU

The Committee for Orphan Medicinal Products (COMP), an arm of the European Medicines Agency (EMA), is recommending that WVE-210201 be designated an orphan drug as a potential Duchenne muscular dystrophy (DMD) treatment, its developer, Wave Life Sciences, announced. WVE-210201 is an investigational compound of a type of therapy called exon skipping, which produces shorter but functional proteins. It is currently in a […]


Catabasis Pharmaceuticals Announces Plans for Edasalonexent Phase 3 POLARIS DMD Trial in Duchenne Muscular Dystrophy

CAMBRIDGE, MA, July 9, 2018 – Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced plans for the Phase 3 POLARIS DMD trial with edasalonexent in patients with Duchenne muscular dystrophy (DMD). Catabasis plans to initiate the global POLARIS DMD trial in the second half of 2018 with top-line results expected in the second […]


Catabasis Pharmaceuticals Presents New Edasalonexent Clinical Biomarker Data Showing NF-kB Inhibition and Target Engagement in the MoveDMD® Trial

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported new NF-kB inhibition biomarker results showing edasalonexent target engagement in the MoveDMD Phase 2 trial and open-label extension in boys affected by Duchenne muscular dystrophy (DMD). NF-kB is a fundamental driver of disease progression in DMD. These results add further support to the significant […]


Early data show Sarepta’s investigational gene therapy led to robust microdystrophin expression in three DMD patients

Shares in Sarepta Therapeutics surged as much as 80 percent Tuesday after the company released preliminary data at an investor event from a Phase I/IIa trial showing that its experimental gene therapy, dubbed AAVrh74.MHCK7, boosted production of a truncated form of a muscle-making protein in three young boys with Duchenne muscular dystrophy (DMD). Specifically, the company reported that all […]


FDA Names Potential FSHD Therapy GBC0905 an Orphan Drug to Speed Its Development

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to GBC0905 as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD),its developer, Genea Biocells, announced. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application fees. GBC0905 […]


CHMP Adopts Positive Opinion for the Expansion of the Translarna™ (ataluren) Label to Include Patients as Young as 2 Years of Age

– European Commission ratification anticipated in coming months – – Approval of the Translarna annual re-assessment also recommended by CHMP – SOUTH PLAINFIELD, N.J., June 1, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval […]


Solid Biosciences Announces New Preclinical Data at the American Society of Gene and Cell Therapy Annual Meeting

– Data Reinforce Potential of SGT-001 as an Important Treatment Candidate for DMD – – Company Continues to Advance Gene Therapy Portfolio – CAMBRIDGE, Mass., May 18, 2018 (GLOBE NEWSWIRE) — Solid Biosciences Inc. (NASDAQ:SLDB) today announced the presentation of new preclinical data from its gene therapy development programs for Duchenne muscular dystrophy (DMD). New […]