CHMP Recommends Renewal of Translarna's™ Marketing Authorization for Nonsense Mutation Duchenne Muscular Dystrophy Based on Continued Positive Benefit-Risk Assessment

November 11, 2016

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 the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients five years and older. In connection with the renewal, the marketing authorization will include a specific obligation to conduct an additional long-term post-authorization trial.

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Capricor Therapeutics Announces Plans to Expand Clinical Development Program in Duchenne Muscular Dystrophy to Evaluate Peripheral and Respiratory Muscle Function

November 10, 2016

Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other serious medical conditions, today announced that it intends to expand its CAP-1002 clinical development program in Duchenne muscular dystrophy (DMD) to encompass the skeletal muscle aspects of the disease, in addition to the cardiac complications.

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cTAP Announces Two Research Publications Categorizing and Predicting Disease Prediction in Duchenne Muscular Dystrophy

October 31, 2016

The Collaborative Trajectory Analysis Project (cTAP), a public-private partnership to accelerate data science solutions to critical problems in drug development for Duchenne Muscular Dystrophy (Duchenne), today announced the publication of two research studies with important implications for the design of effective clinical trials.

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PTC Therapeutics Announces New Analyses that Support the Potential Benefit of Ataluren in Preserving Lung Function in Non-Ambulatory Nonsense Mutation Duchenne Muscular Dystrophy Patients

October 6, 2016

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory nonsense mutation Duchenne muscular dystrophy patients (nmDMD). The results, which are being presented today as part of a company-sponsored symposium, are based on PTC's analyses of lung function data from one of PTC's ongoing open-label extension studies (Study 019) versus natural history data from a comparable non-ambulatory cohort.

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Catabasis Pharmaceuticals Completes Target Enrollment for Part B of the MoveDMD® Trial, a Phase 2 Trial of Edasalonexent (CAT-1004) for the Potential Treatment of Duchenne Muscular Dystrophy

October 4, 2016

Catabasis Pharmaceuticals, Inc. (NASDAQ: CATB), a clinical-stage pharmaceutical company, today announced that target enrollment of 30 patients has been reached for Part B of the MoveDMD trial, a 12-week trial to assess the safety and efficacy of edasalonexent (CAT-1004) in Duchenne muscular dystrophy (DMD). Edasalonexent is an oral small molecule that the Company believes has the potential to be a disease-modifying therapy for DMD patients, regardless of their underlying dystrophin mutation.

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