Catalyst Pharmaceuticals launches Firdapse expanded access program for eligible people living with congenital myasthenic syndromes and Lambert-Eaton myasthenic syndrome
Catalyst Pharmaceuticals, a biopharmaceutical company focused on rare debilitating diseases, announced encouraging results on Sept. 29, 2014, from its phase 3 clinical trial of Firdapse (amifampridine phosphate tablets equivalent to 10mg amifampridine) for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS).
Funding from MDA and three other organizations will allow ReveraGen BioPharma to test its experimental anti-inflammatory drug for Duchenne MD in healthy volunteers
ReveraGen BioPharma, based in Silver Spring, Md., is moving ahead with a phase 1 trial of an experimental compound in development to treat Duchenne muscular dystrophy (DMD), made possible by a $2 million grant from U.S.-based MDA and three United Kingdom-based DMD organizations.
Sarepta Therapeutics continues to report encouraging data from its extension study of eteplirsen, designed to treat Duchenne MD due to specific genetic mutations
Walking ability and respiratory function in boys with Duchenne muscular dystrophy (DMD) show continued benefit from eteplirsen at 144 weeks (almost three years), the drug's developer announced today. In addition, the intravenously infused drug was well tolerated, with no serious treatment-related adverse events seen.
Research and Clinical Trial Updates
What About My Disease?
Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.
An experimental drug designed to reduce scarring in Duchenne MD-affected muscles shows signs of safety and desired activity; FDA will speed its review
HT-100, an experimental drug being developed for Duchenne muscular dystrophy (DMD) with support from MDA, has shown preliminary safety and signs of the desired effects on scar tissue formation in the first 17 trial participants, and it has received "fast track" designation from the U.S. Food and Drug Administration (FDA).
The company plans to submit a New Drug Application to the U.S. Food and Drug Administration later this year, and has committed to conducting two confirmatory post-approval clinical trials
Dutch biopharmaceutical company Prosensa has outlined plans to seek accelerated approval in the United States for its experimental drug drisapersen, under development for the treatment of Duchenne muscular dystrophy (DMD). The company will seek approval for the drug in Europe as well.
Santhera Pharmaceuticals' investigational drug to treat respiratory dysfunction in Duchenne muscular dystrophy has shown benefit compared to a placebo in a phase 3 trial
Results from a phase 3, 65-participant trial of idebenone (brand names are Catena and Raxone) in boys with Duchenne muscular dystrophy (DMD) show the drug reduced the decline in respiratory function compared to a placebo, potentially paving the way toward regulatory approval.