Nitisinone, a drug used to treat rare diseases, can kill malaria-causing mosquitoes, a new study found. In a bid to fight malaria, researchers are coming up with new solutions to reduce the spike ...

Nitisinone was shown to last longer than ivermectin in the human bloodstream, and was able to kill not only mosquitoes of all ages -- including the older ones that are most likely to transmit malaria ...

RNA-based therapeutics are gaining traction due to advancements in delivery technologies, despite historical challenges with ...

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

Childhood-onset striatonigral neurodegeneration robs children of the ability to walk and talk by age five. Researchers at ...

In one tiny town, more than a dozen people were diagnosed with the rare neurodegenerative disease ALS. Why? I n March 2009, ...

Shares of Soleno Therapeutics jumped 40% in morning trading on Thursday, after the company's drug for a rare metabolic condition became the first such treatment to receive U.S. regulatory nod.

The family of nine-year-old Charleigh Pollock will continue to be covered for the drug, which costs $1 million a year, until ...

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

At meeting on human embryo editing, CRISPR pioneer says science is a long way from knowing if germline DNA can be safely ...

He took the stage and was great last week at the Grand Ole Opry’s 100th anniversary show, and now he is down to just two ...

This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal ...