Tinlarebant is the first therapeutic candidate to demonstrate clinical efficacy in Stargardt disease, having met the primary endpoint, reductions ...
Five issued U.S. patents cover the combination of ImmunityBio’s IL-15 receptor agonist (NAI) with Bacillus Calmette-Guérin ...
ENERGY 3 study met 1 of 2 co-primary endpoints in children with ENPP1 deficiency Treatment with BMN 401 led to statistically significant increases in plasma inorganic ...