India, June 4 -- Biogen Inc. (BIIB) announced that salanersen, its novel investigational candidate, was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of Spinal Muscular Atrophy (SMA).
Spinal Muscular Atrophy (SMA) is a rare, genetic, and neuromuscular disease that is caused by a deficiency in the production of survival motor neuron (SMN) protein as a result of a damaged or missing SMN1 gene.
It is characterized by the loss of motor neurons in the spinal cord and lower brain stem that results in progressive muscular atrophy and weakness. SMA affects all age groups and is specifically a leading cause of genetic death among infants.
Salanersen, or BIIB115, is an intrathecally admin...