MDA-funded researchers say SMN, which is deficient in spinal muscular atrophy, is most needed during early life and in skeletal muscle and nerve tissues

posted on February 4, 2014 - 11:11am
Raising levels of the SMN protein, which is deficient in patients with the most common form of spinal muscular atrophy (SMA), has been the holy grail of therapy for this disease since the 1990s and is the goal of several experimental compounds now in development.

The quest to cure spinal muscular atrophy still has many years to go, but the signs of progress are unmistakable

posted on August 29, 2013 - 10:37am
A Closer Look: SMA Slideshow August is often referred to as Spinal Muscular Atrophy (SMA) Awareness Month, but for MDA, work on finding treatments for this degenerative disease — and providing assistance to individuals and families affected by it — is a year-round event. Today, research aimed at slowing, stopping or even preventing spinal muscular atrophy (SMA) is advancing with more speed...

Repligen’s spinal muscular atrophy program, including development of the experimental drug RG3039, now has the support of a major pharmaceutical company

posted on January 4, 2013 - 8:48am
Update (Feb. 2, 2015): Pfizer announced in January 2015 that it will not continue its collaboration with Religen for development of RG3039. The reasons are not clear, but industry observers suspect that the change of plan is related to an announcement in summer 2014 that the compound failed to increase production of the SMN protein.

The trial is designed to evaluate the safety of multiple doses of RG3039 in healthy volunteers and to determine pharmacokinetics (the way the body affects a drug)

posted on October 2, 2012 - 2:45pm
The experimental drug RG3039, being developed by Repligen Corp. of Waltham, Mass., as a potential treatment for spinal muscular atrophy (SMA), has moved into its next phase of testing. RG3039 previously was tested in a phase 1a trial, in which healthy adult volunteers received a single dose of the drug. Now, in a phase 1b trial, healthy volunteers will receive multiple doses of the drug.

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.

Repligen soon will begin a first-ever human trial of a drug created specifically for spinal muscular atrophy;  FDA grants fast track designation

posted on May 20, 2011 - 11:13am
In a historic first, biotech company Repligen Corp., of Waltham, Mass., has received approval from the U.S. Food and Drug Administration (FDA) to begin a phase 1 clinical trial of the experimental drug RG3039 for spinal muscular atrophy (SMA).