The gene-editing technique known as CRISPR is promising to revolutionize medicine. Some researchers are trying to help make it available for people with very rare genetic disorders.

After Natalie Rex was diagnosed with Friedreich’s ataxia -- a rare genetic and usually fatal neurological disease -- her twin ...

Studying patients with PD-1 or PD-L1 deficiency may help inform new approaches to fine-tuning immunotherapies to maximise ...

"The grief spiral and the odyssey of trying to figure out what the problem is takes a really big toll on families." Baton ...

Alzheimer’s disease is a debilitating condition that slowly robs people of their memory and cognitive abilities, affecting millions of people worldwide. Familial Alzheimer’s disease (FAD), a rare ...

The Philippine Health Insurance Corp. (PhilHealth) will expand its health-care packages before the end of the year to cover ...

Arrowhead has entered a worldwide licensing and partnership agreement with Sarepta Therapeutics for rare genetic disease ...

The Delhi high court has directed the Centre and the Delhi government to file comprehensive reports on the availability and ...

In response to the plea filed by several individuals suffering from haemophilia disease, the Delhi High Court has directed ...

Arrowhead Pharmaceuticals has entered a worldwide licensing and partnership agreement with Sarepta Therapeutics to develop treatments for rare genetic diseases. Arrowhead will receive an $825m ...