The U.S. Food and Drug Administration on Wednesday approved Soleno Therapeutics' drug to treat a rare genetic disorder, ...

The FDA Wednesday approved the first drug to treat people with Prader-Willi syndrome, a rare genetic disease that causes an ...

Research shows experimental anti-amyloid drug reduced cognitive symptom risk by 50% in people with rare genetic mutations ...

Researchers studying over 2,000 Ashkenazi Jewish centenarians uncovered two rare IGF-1 gene variants linked to exceptional ...

Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...

In return, Ionis will receive an undisclosed upfront payment from Sobi. It is also eligible to receive milestone payments and a tiered royalty on the drug's future sales.

With drugs on the horizon for treating and possibly preventing transthyretin amyloid cardiomyopathy, cardiologists are ...

That’s why the MINI Act is crucial – it ensures continued investment in GTTs and preserves their potential for nano-rare disorders and other genetic disorders. Generally, rare disease patients ...

P rince Frederik of Luxembourg, the youngest son of Prince Robert of Luxembourg and Princess Julie of Nassau, died in Paris ...

it’s known as early-onset Alzheimer’s disease, which accounts for less than 10% of cases. Early-onset Alzheimer’s can happen ...