Researchers at PTC Therapeutics are looking for participants with Duchenne muscular dystrophy to participate in a Phase 3 study. The goal of the study is to evaluate the safety and efficacy of Translarna (ataluren). The drug is believed to promote the formation of full-length and functional dystrophin protein in boys with a nonsense (or premature . . .
Like many fire fighters, Brian Glenn, Battalion Chief – EMS in the Denton Fire Department, was aware of MDA because of the International Association of Fire Fighter’s long commitment to raising money for treatments and cures for muscular dystrophy and related diseases. Since 1954, fire fighters have been asking their community to “fill the boot” . . .
The Muscular Dystrophy Association recently awarded an MDA human clinical trial grant for the development of a critical biomarker for Charcot-Marie-Tooth disease (CMT) to Mary M. Reilly, professor of clinical neurology and consultant neurologist, UCL Institute of Neurology and National Hospital for Neurology and Neurosurgery, London. The investment, totaling $1 million over three years, will . . .
Former NFL great Dwight Clark died from ALS (amyotrophic lateral sclerosis) on June 4, a little more than one year after revealing he had the disease. Clark was immortalized when he caught the winning touchdown pass in the NFC Championship Game in January 1982, sending the San Francisco 49ers to their first Super Bowl. The . . .
This morning, Summit Therapeutics announced that it is discontinuing development of ezutromid for Duchenne muscular dystrophy after primary and secondary endpoints were missed after 48 weeks of treatment in their PhaseOut DMD trial. PhaseOut DMD was a phase 2, multi-center, open-label clinical trial of ezutromid, a utrophin-modulation therapy. Thirty-eight boys enrolled in the trial completed the 48-week regimen. The . . .
This week, Sarepta Therapeutics announced encouraging preliminary results from a phase 1/2a gene therapy trial designed to assess the investigational drug AAVrh74.MHCK7.micro-Dystrophin in boys with Duchenne muscular dystrophy (DMD). After 90 days in the trial, the first three participants all showed robust expression of micro-dystrophin — a shortened version of the protein that is absent . . .