Studying the mechanisms controlling gene activity
Slip the word “genetics” into a casual conversation and you likely won’t get too many questioning looks.
Most people basically understand that “genetics” refers to DNA and genes, the blueprint of life, the code that determines whether someone has blue eyes or green. Many are familiar, too, with the idea that “genetics” can cause a variety of health conditions: diabetes, breast cancer, muscular...
Repligen Corp. is proceeding with development of an experimental compound to treat Friedreich's ataxia, with help from a new MDA grant.
Development of a promising experimental medication to treat Friedreich's ataxia (FA) is proceeding, with help from a $731,534 grant MDA awarded to Repligen Corp. of Waltham, Mass., this month (December 2009).
This is the second research grant that MDA has awarded to the small biopharmaceutical company, through the Association's translational (laboratory-to-clinic) research program.
A compound being developed to treat Friedreich's ataxia specifically targets an unwanted molecular brake
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
This article presents reports on research in: Duchenne MD, Becker MD, facioscapulohumeral MD, limb-girdle MD types 2A and 2B, Myioshi myopathy, nemaline myopathy, polymyositis, dermatomyositis, inclusion-body myositis, slow-channel myasthenic syndrome, myasthenia gravis, Lambert-Eaton syndrome and spinal muscular atrophy.
Research progress in spinal muscular atrophy (SMA) has been considerable in recent months.