Diseases of the Peripheral Nerves

BioMarin has purchased the rights to Repligen’s histone deacetylase (HDAC) inhibitor portfolio, including the experimental FA drug RG2833, whose development MDA has supported

posted on January 22, 2014 - 3:01pm
Experimental Friedreich's ataxia (FA) drug RG2833, which has been in early-stage development by Massachusetts company Repligen with support from MDA, is now owned by California biotechnology company BioMarin.

BioMarin has purchased from Repligen the rights to experimental compounds for Friedreich’s ataxia and other neurological disorders

posted on January 21, 2014 - 2:39pm
Update (Jan. 22, 2014): Read FA: BioMarin To Develop Former Repligen Drugs for an updated version of this story.

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

posted on July 12, 2013 - 11:05am
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

posted on June 27, 2013 - 10:15am
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

posted on June 4, 2013 - 11:49am
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

posted on May 17, 2013 - 4:39pm
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

posted on March 1, 2013 - 5:00am
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.

MDA-supported researchers report that mice with a disorder resembling type 1B Charcot-Marie-Tooth disease benefited from treatment with curcumin, a component of the spice turmeric

posted on January 21, 2013 - 5:00am
Mice with a disorder resembling the type 1B form of Charcot-Marie-Tooth disease (CMT) benefited from treatment with either of two forms of oral curcumin, a component of the spice turmeric, according to researchers supported in part by MDA.