Boys with Duchenne muscular dystrophy between the ages of seven and 16 are now being recruited to participate in a phase 3 clinical trial to evaluate the effectiveness and safety of eteplirsen in treating DMD.
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).
Cambridge, Mass.-based biotechnology company Sarepta Therapeutics updated the Duchenne muscular dystrophy (DMD) community about its pipeline of drugs in development for this disorder via an Aug.
A phase 2 clinical trial of the drug rituximab (Rituxan) in adults with myasthenia gravis (MG) is now open at 15 U.S. centers with plans for additional sites.
Rituximab suppresses a specific part of the body's immune system and is approved by the U.S. Food and Drug Administration (FDA) to treat rheumatoid arthritis and other disorders.
The investigational oral drug ataluren, in development to treat Duchenne muscular dystrophy (DMD) resulting from a specific type of genetic mutation, has received conditional approval in the European Union (EU). This designation means patients can receive it in the EU and EU-associated countries while additional studies are being conducted.
California-based Isis Pharmaceuticals has announced the opening of a phase 3 clinical trial of its experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA), a muscle-weakening disease that results from loss of nerve cells in the spinal cord.
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.