Tikvah Receives Orphan Drug Status Spinal Muscular Atrophy Treatment

Tikvah Therapeutics reported that the FDA has granted orphan drug status to sodium phenylbutyrate for the treatment of spinal muscular atrophy (SMA).

SMA is a group of inherited and often fatal diseases that destroy the nerves controlling voluntary muscle movement, affecting crawling, walking, head and neck control and swallowing.

There currently are no approved therapies for the treatment of SMA.