Isabella Brown has been diagnosed with Turner Syndrome, which affects around one one in 2,000 births ...

BioMarin will discontinue dosing and enrollment of its phase 2 trials for Voxzogo—currently approved in achondroplasia, a ...

Non-invasive prenatal testing (NIPT) is a simple blood test during pregnancy that helps detect rare genetic conditions early, ...

With Ascendis Pharma entering the achondroplasia space last month and BridgeBio on deck, BioMarin faces competition. Adding ...

One minute, an actor is everywhere — dominating the box office, making magazine covers, popping up on late-night shows — and the next, they’re gone.Whether it’s someone like Ke Huy Quan, who struggled ...

Some actors take years off, only to return when the right role lands in their lap. Others simply step away from the spotlight ...

Prader-Willi syndrome (PWS) is a rare genetic condition marked by uncontrolled hunger and complex behavioral and medical ...

A deep dive into the iconic late-night block that shaped the tastes of a bleary-eyed, post-ironic generation of comedy fans.

A lawsuit alleging Bristol, Tennessee, police and paramedics subjected a man having a seizure to excessive force and failed ...

Life-saving treatments for children with orphan illnesses often move slowly, if at all, through the federal approval process.

Parents of 6-year-old Raiden Pham say new FDA feedback brings hope as they work to raise $2 million for future gene therapy, ...

For Mary Sims, the nightmare began with what she thought was a simple case of the flu. Instead, it spiraled into a grueling ...