by Peter Ciszewski | Jul 21, 2017
Spark Therapeutics’ announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the U.S. Food and Drug Administration (FDA) had granted rare pediatric disease designation to Luxturna (voretigene neparvovec), a gene therapy currently...
by Peter Ciszewski | Jul 14, 2017
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Mallinckrodt for their potential Duchenne muscular dystrophy (DMD) treatment, MNK-1411 (a long-acting depot formulation of tetracosactide, a melanocortin receptor agonist). DMD is a...
by Peter Ciszewski | Jul 13, 2017
The U.S. Food and Drug Administration on Friday approved the first new drug for sickle cell disease in nearly 20 years. Endari (L-glutamine oral powder) helps reduce severe complications associated with the blood disorder, the agency said. “Endari is the first...
by Peter Ciszewski | Jul 10, 2017
Cytokinetics (located in South San Francisco) is a late-stage biotechnology company developing first-in-class muscle activators as potential treatments for debilitating diseases in which muscle performance is compromised and/or declining. This emerging biotech company...
by Peter Ciszewski | Jun 28, 2017
A new study shows that “Genome sequencing” of healthy people reveals that some are at risk for rare genetic diseases. The researchers said doctors need to be sensitive when revealing that information. Study lead author Dr. Jason Vassy said,...
