In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

The US Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time treatment that proved to be life-changing for a small number of children in a ...

In LAMA2-related muscular dystrophy, weakened extracellular matrix (yellow) leads to muscle fiber degeneration and the infiltration of immune cells (red) into the tissue. Researchers at the University ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Advancements in genetics have already begun changing the way scientists study, predict, diagnosis and treat diseases. But how does the public feel about gene therapy and gene editing? In general, the ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

Genetic diseases impact almost 70-80 million people worldwide. Oftentimes, there are limited treatments that doctors can provide, leaving patients with few interventions to manage symptoms. Recently, ...

Five-year clinical study results show this revolutionary one-time treatment allowed 94% of patients to stop routine factor IX prophylaxis "I'm just very blessed to be in the right spot at the right ...

Physician and molecular biologist Katherine High remembers sitting at a staff meeting of the gene therapy company Spark Therapeutics on November 15, 2018, waiting to hear from a guest speaker, when ...

When Santa Claus brought 10-year-old Kathy High a junior chemistry set for Christmas more than 60 years ago, he helped spark a lifelong interest in science, and today the North Carolina native — now ...