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

With a storied history in vaccine development, India's Bharat Biotech is shaking things up and answering the call of advanced ...

Medical history has recently been made in a New York City hospital. This month, 21-year-old Sebastien Beauzile became the ...

SineuGene Therapeutics Co., Ltd. ('SineuGene'), a clinical-stage biotech company pioneering innovative therapies for neurological disorders, today announced that the U.S. Food and Drug Administration ...

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

The company expects next quarter to read out initial data from its Heart-2 trial, which is testing a one-time treatment for people with genetically elevated cholesterol.

Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating ...

A new study in the journal Human Gene Therapy indicates that DNA impurities derived from plasmid and host cell DNA are ...

Resilience relies on the brain’s ability to regulate emotion, adapt, and recover. Myelin plasticity helps speed up and stabilize resilient brain responses. Genes like FKBP5 and BDNF affect how people ...

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

Wu Kaimin was worried her dissertation proposal might not be approved. Now, her research could fundamentally alter how ...

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