Gabriella Ngang, of Farmington Hills, is one of three in Michigan to take part in a clinical trial of a new sickle cell ...

The GOP tax bill repeals the Inflation Reduction Act’s price controls for drugs for rare diseases.

Using an AI-driven drug discovery process, the drug known as vorinostat was identified as a promising treatment for Rett ...

A recent study involving researchers from the University of Basel reveals that slowing down the intracellular transport of ...

Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are ...

Scientists discovered a cancer-fighting compound in mold from ancient tombs, revealing a powerful new drug against leukemia.

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

The newly licensed drug, leniolisib, is the first targeted treatment for her condition, Activated PI3-kinase Delta Syndrome ...

HealthDay News — Drugmaker Eli Lilly plans to buy Verve Therapeutics, a gene-editing startup, for about $1 billion upfront. The deal gives Lilly a potential new treatment for heart disease, The Wall ...

Genetically engineered skin grafts exhibited a long-term ability to heal large, chronic wounds in patients with recessive ...

Ferring Pharmaceuticals officially opened doors to its state-of-the-art gene therapy manufacturing facility in Parsippany ...

(Reuters) -The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute ...