Positive Preclinical Proof-of-Concept Results For Mitochondrial Protein Replacement Platform In Friedreich's Ataxia
Wednesday, April 1, 2015 - 17:40
in Health & Medicine
BioBlast Pharma Ltd. has announced positive preclinical in vitro and in vivo proof-of-concept study results for its mitochondrial protein replacement therapy drug candidate (BB-FA) for Friedreich's Ataxia. Friedreich's Ataxia is an inherited disorder characterized by progressive deterioration of the muscular and nervous system that begins in the first or second decade of life and results in gait disturbance (ataxia), cognitive impairment, progressive heart disease and diabetes. According to Friedreich's Ataxia Research Alliance (FARA), about 1:50,000 people in the U.S. suffer from Friedreich's Ataxia. Most patients are wheelchair-bound within 15 years of diagnosis. read more