ENDPOINTS NEWS15m
Re­genxbio shares first da­ta show­ing Duchenne gene ther­a­py im­proves mus­cle func­tion
Regenxbio reported improved muscle function in boys with Duchenne muscular dystrophy using their gene therapy RGX-202. The ...
ENDPOINTS NEWS36m
As car­diomy­opa­thy mar­ket ex­pands, Al­ny­lam of­fers look at Phase 1 da­ta for new drug
Alnylam's nucresiran showed promising results in Phase 1 trial for rare cardiomyopathy, achieving up to 96% TTR reduction at ...
ENDPOINTS NEWS7m
A rare neu­ro­log­i­cal dis­ease is Sage’s last, best hope af­ter fail­ures in de­pres­sion
In 2010, neuroscientist and former Eli Lilly research leader Steve Paul started a company that he hoped would spark a revolution in drug discovery built around a class of compounds dubbed ...