Stable vision and retinal anatomy for up to 5 years, no supplemental therapy for up to 2 y ...

Gene therapy has changed what is possible for people living with sickle cell disease. New treatments have shown that the ...

Rare-disease families can now invest in Mahzi Therapeutics' gene therapy, giving patients a financial stake in developing ...

Learn how gene editing is transforming sickle cell disease treatment and why expanding access to younger children matters.

Over the last five years, Canadian ALS clinics have shifted from offering genetic testing mainly to people with a family ...

A gene therapy designed to replace the missing protein that causes fragile X syndrome restored several disease-relevant ...

The Ara Parseghian Medical Research Fund will provide financial backing for the multi-year research effort.

A gene therapy that instructs cells to produce more of an anti-ageing protein called klotho is about to be offered by a US company at overseas clinics to bypass FDA rules ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

Epilepsy affects more than 50 million people worldwide, making it one of the most common neurological disorders. Although ...

Daniel Cressy was diagnosed with sickle cell disease as a child. Thanks to a newly approved gene therapy, on May 22, Cressy ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...