FA is a progressive neuromuscular disease that causes loss of coordination and mobility. My parents got my test results the ...

New research demonstrates the potential of precise genome editing technologies, called adenine base editors, to correct disease-causing mutations in stem cells from patients with X-linked chronic ...

"The unique mechanism we've identified here suggests that there are more long non-coding RNAs underlying rare genetic ...

Researchers found that ultra-rare genetic variants play a significant role in the heritability of coronary artery disease ...

For Lisa Hawker, getting a diagnosis of her daughter Jaydi's rare genetic condition was life-saving. Jaydi has a rare form of ...

Nager syndrome, or NS, is a rare genetic disease that affects the development of the face and limbs, usually causing anomalies in the bone structures of the jaws, cheeks, and hands. With a ...

Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, ...

Sidra Medicine s Genetic and Genomic Medicine clinic is providing life altering gene therapy for rare genetic disorders. In a ...

A TEEN has spent the last 10 years in a wheelchair after he was diagnosed with a rare genetic muscular disease known as ...

The former prime minister said the cause is close to his heart, having lost his son Ivan to a rare genetic disease 15 years ...

At #STATSummit, three parents discussed the challenges they've faced trying to find treatments for their children with rare ...