Sebastien Beauzile, 21, is the first New Yorker to have received the breakthrough Lyfgenia treatment, according to the New ...

Shares of Sarepta Therapeutics SRPT plunged nearly 24% last week after the company reported the death of a patient following ...

The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development.

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

CRISPR technique can target single genes essential for cancer cell survival, toppling the whole structure, says nanomedicine ...

By John Lauritsen Click here for updates on this story Minnesota (WCCO) — An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

July 17, 2024 — A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic ...

Treatment shows potential to slow the progression of human degenerative eye diseases, including retinitis pigmentosa.

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Beam Therapeutics reported promising Phase 1/2 data for BEAM-302, demonstrating durable gene correction for AATD. See why I ...

A research team led by Sheri Holmen, Ph.D., investigator at Huntsman Cancer Institute and professor in the Department of ...