PTK-SMA1

posted on December 31, 2009 - 1:17am
Featured in this issue: Duchenne and Becker MD ** Facioscapulohumeral MD ** Friedreich's ataxia ** Limb-Girdle muscular dystrophy ** Myotonic dystrophy ** Spinal muscular atrophy

Researchers have identified a compound that helps cells produce more full-length SMN protein from the backup SMN2 gene.

posted on November 6, 2009 - 1:19pm
Scientists have identified a chemical cousin of the commonly used antibiotic tetracycline that has the potential to be refined and modified into a therapy for spinal muscular atrophy (SMA). PTK-SMA1 works by correcting an error in a cellular process called RNA splicing, and leads to increased production of a critical protein that is deficient in this disease.