Research and clinical trial updates
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...
Research updates and clinical trials information
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.
A phase 1 gene transfer trial in nine babies with type 1 spinal muscular atrophy has opened at Nationwide Children's Hospital in Columbus, Ohio
A phase 1 trial to test the safety and efficacy of gene transfer therapy in infants with type 1 spinal muscular atrophy (SMA) who are 9 months old or younger has opened at Nationwide Children's Hospital in Columbus, Ohio, under the direction of neurologist Jerry Mendell.
Preliminary results for a phase 2 trial of the experimental compound olesoxime suggest it may preserve motor function in spinal muscular atrophy
Update (Feb. 2, 2015): Swiss pharmaceutical company Roche announced in a Jan. 16, 2015, press release, that it will acquire Trophos and continue development of olesoxime for SMA. "We will build on the work done by Trophos and the French Muscular Dystrophy Association to advance the development of olesoxime and to bring it to people who with live with this devastating condition as quickly as...
Preliminary results from four infants in a phase 2 trial of ISIS-SMNRx suggest the drug is well-tolerated and may prolong ventilator-free survival
Interim results of a phase 2 clinical trial to test multiple doses of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA) suggest that the drug is well-tolerated and may prolong ventilator-free survival.
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
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.
A spring 2014 course from Ohio State University will emphasize Duchenne muscular dystrophy mechanisms, research, and clinical and social aspects
A course on Duchenne muscular dystrophy (DMD), geared toward graduate and upper-division undergraduate students and held at Ohio State University College of Medicine and Nationwide Children's Hospital in Columbus, is open to the public, without charge, via real-time streaming and podcasts.