Why didn’t analysts share the same fear that gripped investors about Sarepta stock? One reason is the known risk of acute liver injury associated with adeno-associated virus vector (AAV)-based gene ...

Things are going from bad to worse for Sarepta Therapeutics (NASDAQ: SRPT), a biotech focusing on gene therapies. The company ...

This collection of health news showcases a range of significant developments: measles cases are surging in Texas and New ...

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

Recent health updates include a rise in measles cases in Texas and New Mexico, expansion of Alnylam's heart disease drug ...

Circadian rhythms are endogenous time-keeping mechanisms that regulate physiological processes in response to external light-dark cycles.

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 ...

Alnylam's Amvuttra enters ATTR-CM drug race, and Expedition Therapeutics seeks to bring Chinese drugs to the U.S.

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

Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...