The Icahn School of Medicine at Mount Sinai has been awarded a grant of more than $4 million from The Leona M. and Harry B. Helmsley Charitable Trust to support an innovative research project aimed at understanding the early stages of Crohn’s disease before noticeable symptoms develop.
Regenxbio gives glimpse at early Duchenne gene therapy results, plans pivotal study for accelerated approval
Regenxbio shared early biomarker results from a 12-year-old boy with Duchenne muscular dystrophy who received a higher dose of its gene therapy, and the biotech