The growing prevalence of ultrahigh-cost drugs under development is prompting health systems nationwide to rethink strategies ...

Hubble Therapeutics LLC a patient-focused biotechnology company developing a gene therapy to cure pediatric blindness, announces the successful ...

Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, discusses data supporting the FDA approval of revakinagene taroretcel-lwey (Encelto; Neurotech Pharmaceuticals), the first and only therapy ...

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.

The company’s Phase I trial found that patients remained disease free after three months utilising the lowest doses of RAG-01 ...

Verve received FDA IND clearance for VERVE-102 to treat HeFH, adding new trial sites in the United States. Read more about ...

This infographic explores the role of gene therapy in treating haemophilia, focusing specifically on haemophilia A and B. It ...

A team of scientists from China has made a breakthrough in understanding how aging affects muscles and found a possible way ...

The arc of biotechnology is one of breakthroughs and struggles, writes Tim Hunt, CEO of the Alliance for Regenerative ...

The goal of genome editing technology is to develop strategies for the correction of those mutations that are responsible of ...

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 ...

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