Quest News (online only)

Isis Pharmaceuticals updated the SMA community on phase 2 clinical trial results of the drug ISIS-SMNRx.

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:

Boys between the ages of seven and 16 with Duchenne muscular dystrophy are being recruited to participate in a phase 3 clinical trial to evaluate the effectiveness and safety of eteplirsen in treating DMD.

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.

Catalyst Pharmaceuticals launches Firdapse expanded access program for eligible people living with congenital myasthenic syndromes and Lambert-Eaton myasthenic syndrome

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).

Sarepta Therapeutics updated the Duchenne MD community on regulatory and clinical trial progress with respect to eteplirsen, SRP-053 and SRP-045 on Aug. 7, 2014

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.

A phase 2 clinical trial of rituximab in myasthenia gravis has now opened at 15 U.S. centers under the auspices of NIH's NeuroNEXT

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.

Duchenne patients in the European Union with specific dystrophin gene flaws can receive the drug ataluren prior to its full approval

posted on August 4, 2014 - 2:44pm
Update (Oct. 24, 2014): Enrollment for the phase 3 trial of ataluren is complete, with results expected in the second half of 2015. See the PTC press release of Sept. 9, 2014. ===================================================================================== original story:

Isis Pharmaceuticals is testing its experimental, antisense-based drug in a phase 3 trial in SMA-affected babies age 7 months or less

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.