drug testing

In a phase 1 trial in healthy volunteers, Repligen's experimental therapy for spinal muscular atrophy was safe and well-tolerated

posted on April 25, 2012 - 4:00am
Update (June 21, 2012): This story was updated to include the fact that MDA funding helped support the phase 1 trial, as well as the preclinical development of RG3039.

The experimental drug SMT C1100 is designed to increase utrophin production as a treatment for Duchenne MD

posted on April 24, 2012 - 4:00am

A phase 1 clinical trial has begun in Turin, Italy, to test the safety of RG2833 in adults with Friedreich's ataxia

posted on March 15, 2012 - 11:00am
The biopharmaceutical company Repligen Corp., headquartered in Waltham, Mass., has launched a phase 1 clinical trial to test its experimental compound, RG2833, in adults with Friedreich's ataxia (FA). The trial is the first of a therapy specifically developed to treat the underlying molecular cause of FA. The phase 1 trial is being conducted at San Luigi Gonzaga University Hospital in Turin,...

MDA has awarded $750,000 to Summit Corp. to test a new formulation of its utrophin booster SMT C1100 for Duchenne muscular dystrophy

posted on December 20, 2011 - 12:05pm
MDA has awarded $750,000 to Summit Corporation PLC for development and testing of SMT C1100, the company's experimental drug for treatment of Duchenne muscular dystrophy (DMD). Summit is an Oxford, United Kingdom, drug discovery company. The award was made through MDA's Venture Philanthropy (MVP) arm, a part of MDA's translational research program.

An MDA-supported open-label trial found the asthma drug benefited 17 of 18 people with either of two forms of congenital myasthenic syndrome

posted on November 9, 2011 - 2:56pm
Researchers have found that the drug albuterol appears to be beneficial in two forms of congenital myasthenic syndrome (CMS) — CMS related to mutations in the collagen Q (colQ) gene and CMS related to mutations in the DOK7 gene.

Clinical trial testing experimental enzyme replacement drug in people with Pompe disease who are at least 13 years old

posted on November 8, 2011 - 10:08am
Update (March 27, 2013): BioMarin announced March 19, 2013, that it plans to move to a phase 2/3 trial of this drug for late-onset Pompe diseae, pending a review by regulatory authorities. See Pompe disease: Late-onset treatment moves forward.