Research

posted on October 17, 2014 - 8:00am
Isis Pharmaceuticals, developer of the experimental spinal muscular atrophy (SMA) drug ISIS-SMNRx, revealed positive results from phase 2 clinical studies of the drug focusing on infants and children with SMA. The new data, which was shared on October 10 by ISIS representatives attending the 19th International World Muscle Society Congress in Berlin, included the following highlights:
posted on October 7, 2014 - 8:20am
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.
posted on October 3, 2014 - 2:00pm
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).
posted on August 11, 2014 - 10:15am
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.
posted on August 11, 2014 - 8:18am
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.
posted on August 4, 2014 - 2:44pm
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.
posted on August 1, 2014 - 10:24am
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.
posted on July 17, 2014 - 8:00am
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.