Maria Lowe, Ph.D., associate vice president of Pharmaceutical Intelligence at Institute for Clinical and Economic Review ...

Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...

It expects that any patent from this application will expire in 2038, excluding any potential patent term extension or ...

Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle diseases and blood disorders, requires efficient and specific delivery of the ...

A recent study led by the UAB demonstrates the potential of gene therapy to restore motor capacity in an ultra-rare disease, ...

A recent study demonstrated the potential of gene therapy to restore motor capacity in an ultra-rare disease, megalencephalic ...

A recent study led by the UAB demonstrates the potential of gene therapy to restore motor capacity in an ultra-rare disease, ...

To help improve gene therapy, a multidisciplinary team has generated a comprehensive atlas that researchers can use to select ...

Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved ...

In response to a request from the European Medicines Agency (EMA) following the death of a 16-year-old patient in the U.S., ...

U.S. IND clearance follows recent approval of CTA for EPI-321 in facioscapulohumeral muscular dystrophy (FSHD) in New Zealand ...