In its Summer 2013 round of grants, MDA awarded 31 new grants totaling $8.5 million.
Research progress in one disease can lead to progress in other diseases — and MDA’s latest round of grants seek to leverage this potential
In its summer 2013 round of research grant awards, the Muscular Dystrophy Association aims to catalyze research progress in a dozen neuromuscular diseases, with an eye toward applying that knowledge to related muscle diseases, as well.
“A large number of our grants are investigating new therapeutic technologies,” notes Jane Larkindale, MDA's vice president of research. “These are 'platform'...
People with FA, or other disorders classified as ataxias, are encouraged to enroll in the CoRDS ataxia patient registry, which aims to advance ataxia research
The Coordination of Rare Diseases at Sanford (CoRDS) national rare disease registry is now hosting an ataxia patient registry for people with Friedreich's ataxia (FA) or other disorders classified as ataxias (conditions that cause problems with balance or coordination).
Four grams a day of vitamin C wasn't beneficial in type 1A Charcot-Marie-Tooth disease, but the trial yielded advances in CMT trial design
An MDA-supported trial of high-dose ascorbic acid (vitamin C) in the type 1A form of Charcot-Marie-Tooth disease (CMT) (CMT1A) did not find a benefit for this treatment, although it appeared safe and was generally well-tolerated. There were no serious adverse events judged to be related to the study drug.
Nighttime respiratory problems may be more common in people with Friedreich's ataxia than in the general population, necessitating the need for regular screening
Results from a study conducted in Australia show that a nighttime respiratory problem called obstructive sleep apnea occurs more frequently in people with Friedreich's ataxia (FA) than in the general population.
Jim Rusche from Repligen Corp. announced encouraging interim results from a phase 1 clinical trial of RG2833 in Friedreich's ataxia
Interim results from a phase 1 clinical trial of RG2833 in people with Friedreich’s ataxia (FA) show that the experimental drug is well-tolerated, and that it appears to increase the activity of the gene for the frataxin protein.
Data has failed to confirm that treatment with Catena is beneficial in Friedreich's ataxia; sales of the drug in Canada will be discontinued April 30, 2013
Idebenone (under the brand name Catena) in July 2008 received conditional market approval in Canada for the treatment of Friedreich's ataxia (FA). Now, based on additional data that fails to confirm that treatment with Catena is beneficial in FA, Santhera Pharmaceuticals has announced it will discontinue sales of the drug April 30, 2013.