Spinal Muscular Atrophy - SMA

Research and clinical trial updates

posted on July 14, 2015 - 3:03pm
Powerful Research MDA welcomes a neurological disease expert to the family

Research and clinical trial updates

posted on April 21, 2015 - 9:03am
Quest Spring 2015
Research Leadership MDA welcomes two new muscle disease and ALS experts As part of MDA’s bold plan to accelerate treatments and cures for neuromuscular diseases, two new scientific program officers have joined the MDA family: Amanda Haidet-Phillips, Ph.D., and Laura Hagerty, Ph.D. Under the leadership of MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D., they will...
The experimental antisense drug will be tested in infants with a genetic spinal muscular atrophy diagnosis who are to date without symptoms
posted on March 16, 2015 - 11:03am
Biogen Idec has announced plans to run a phase 2 clinical study of the experimental drug ISIS-SMNRx  in infants with spinal muscular atrophy (SMA)who are less than 6 weeks old and have a genetic diagnosis of the disorder but are not yet showing symptoms. 

Stanford University is collecting samples of blood, muscle and other tissues from people with neuromuscular disorders for use in research

posted on January 7, 2015 - 12:06pm
Ever wondered how someone with a neuromuscular disorder in his or her family might contribute to research efforts in this field?

Research updates and clinical trials information

posted on January 5, 2015 - 9:07am
Quest Winter 2015
What About My Disease? Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.

Isis Pharmaceuticals has opened a second phase 3 trial to test its "antisense" drug for spinal muscular atrophy in children ages 2 to 12

posted on November 21, 2014 - 10:19am
Update (Dec. 1, 2014): Isis has announced that its partner, Biogen Idec, plans to conduct a phase 2 trial of ISIS-SMNRx in up to 25 presymptomatic newborns genetically predisposed to develop SMA, as well as a phase 2 study of this drug in approximately 20 patients with infantile or childhood-onset SMA who do not meet the inclusion criteria for the current phase 3 studies. These two additional...

The first three infants in a gene therapy trial for spinal muscular atrophy have been treated; the trial continues, and additional trials are planned

posted on November 18, 2014 - 9:24am
Dallas-based biotechnology company AveXis, which is developing gene therapy for spinal muscular atrophy (SMA), announced in October that administration of its experimental gene transfer compound to the first three patients in a

Research updates and clinical trials information

posted on October 30, 2014 - 9:01am
Quest Fall 2014
What About My Disease? Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.