Individuals with ALS and their families find unwavering support from MDA
Like all people living with amyotrophic lateral sclerosis (ALS), Joan Sucher, 65, a mother of three adult children in Ballwin, Mo., was devastated when she received her diagnosis. “My life collapsed,” Sucher shares in an email interview with Quest. “We were inconsolable. I had been healthy and active all my life. I ran regularly and finished nine half marathons after age 48. And I had no family...
Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline
Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014.
PTC is moving forward with ataluren
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.
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 European Medicines Agency expressed a 'positive opinion' on conditional approval for ataluren (Translarna) to treat Duchenne MD; a decision is expected within three months
The experimental Duchenne muscular dystrophy (DMD) drug ataluren (which has been given the brand name Translarna) has received encouragement from the European Medicines Agency (EMA) for conditional approval in European Union countries.