A gene therapy from Regeneron Pharmaceuticals Inc. improved hearing in nearly all deaf children in a clinical trial, results ...

OTO, is one of several early-stage gene therapies being developed to treat relatively straight-forward causes of genetic ...

Regeneron Pharmaceuticals said on Monday its experimental gene therapy showed improved hearing in 10 of 11 children between ...

In January 2025, various US Food and Drug Administration (FDA) centers and offices published their guidance agendas for the 2025 calendar year ...

Neurogene Inc. (NASDAQ: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neu ...

"We have, for the first time, an effective treatment for the most severe form of childhood blindness, and a potential ...

Allogene Therapeutics published new long-term follow-up data on cemacabtagene ansegedleucel, showing the investigative ...

Miltenyi Biotec launched India's first Cell and Gene Therapy Center in Hyderabad. The Miltenyi Innovation and Technology ...

MeiraGTx is advancing its investigational gene therapy toward expedited approval in the UK after 11 children who were born ...

Strong evidence for ALS often comes from familial forms of the disease, where specific gene mutations— for example in the ...

Democrats have long opposed honoring Thomas, a Georgia native who was accused of sexual harassment during his 1991 confirmation hearing.

In recent weeks, Children’s Hospital Los Angeles in East Hollywood has taken steps to improve care for some of its pediatric patients. In late January, ...