News

Pfizer shares update on Phase II Clinical Trial with Canadian sites recruiting

According to DuchenneConnect, Pfizer has shared updates on the Phase II clinical trial of the investigational compound, Domagrozumab, a myostatin inhibitor, in people with Duchenne.  According to the update, there are Canadian locations recruiting for the trial in Calgary, Vancouver, London and Montreal.   To see the sites, visit the clinicaltrials.gov page here.

For more information, check out the latest update to the Domagrozumab (PF-06252616) FAQ sheet on DuchenneConnect.

Summit Enrolls Patients in the US into PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid

Summit Enrolls Patients in the US into PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid

Summit Therapeutics, today announced that it has enrolled its first patients at trial sites in the US into PhaseOut DMD, a Phase 2 proof of concept clinical trial of ezutromid in patients with DMD. Ezutromid dosing is expected to follow within a screening period of up to 28 days. Enrolment and dosing of patients into PhaseOut DMD in the UK is ongoing. Ezutromid is a utrophin modulator and represents a potential disease modifying treatment for all patients with DMD.

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CHMP Recommends Renewal of Translarna’s™ Marketing Authorization for Nonsense Mutation Duchenne Muscular Dystrophy

CHMP Recommends Renewal of Translarna’s™ Marketing Authorization for Nonsense Mutation Duchenne Muscular Dystrophy

PTC Therapeutics, Inc. 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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cTAP Announces Two Research Publications Categorizing and Predicting Disease Progression in DMD

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 (DMD), today announced the publication of two research studies with important implications for the design of effective clinical trials.

All patients with Duchenne eventually lose the ability to walk, but the rate at which ambulatory function declines can vary greatly between patients. This variability can cloud interpretation of clinical trials making it difficult to discern whether or not a drug is effective. The published studies announced here explain approximately half of the variability in disease progression in Duchenne, more than double that explained previously with conventional analyses.

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