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.

In Friedreich's ataxia research mice, treatment with interferon gamma preserved sensory neurons, improved muscle function and coordination, and enhanced the ability to move

posted on June 27, 2012 - 2:30pm
Treatment with an engineered version of the naturally produced interferon gamma protein enhanced the ability to move, and improved balance and coordination in mice with a disease resembling Friedreich's ataxia (FA), a team of researchers has reported. In addition, treatment with interferon gamma prevented degeneration of sensory neurons (nerve cells) in the mice.

Precision delivery of human frataxin protein into cells favorably affects growth, life span and the heart in a mouse model of Friedreich’s ataxia

posted on December 26, 2011 - 6:00am
A research team has demonstrated that a cell-penetrating molecule called TAT transported human frataxin protein to its proper place in cells, where it normalized growth, improved heart structure and function, and increased survival rate and life span in a mouse model of severe Friedreich’s ataxia (FA).
posted on January 1, 2011 - 2:20pm
QUEST Vol. 18, No. 1
From childhood science classes, you may remember the periodic table listing all the known chemical elements. You might also recall, in approximately the center of the table, the symbol “Fe” signifying the metal iron. Produced inside large stars in distant space, iron is the most common element on Earth. From earliest times, humans have used it in everything from paint pigments, cookware and tools...

News on amyotrophic lateral sclerosis, congenital muscular dystrophy, Friedreich's ataxia and spinal muscular atrophy

posted on August 27, 2010 - 11:20am
Amyotrophic lateral sclerosis (ALS)
posted on December 31, 2009 - 1:17am
QUEST Vol. 17, No. 1
Featured in this issue: Duchenne and Becker MD ** Facioscapulohumeral MD ** Friedreich's ataxia ** Limb-Girdle muscular dystrophy ** Myotonic dystrophy ** Spinal muscular atrophy

A compound being developed to treat Friedreich's ataxia specifically targets an unwanted molecular brake

posted on September 29, 2009 - 11:32am
Scientists at the Scripps Research Institute in La Jolla, Calif., and the Repligen Corporation in Waltham, Mass., have identified the precise biochemical brake that limits production of a needed protein in Friedreich's ataxia (FA) and determined that this brake is specifically targeted by an experim
posted on November 1, 2008 - 11:09am
QUEST Vol. 15, No. 6
Story includes research items about: limb-girdle and Duchenne muscular dystrophies, myasthenia gravis, mitochondrial myopathy, Friedreich's ataxia, spinal muscular atrophy