Two families from Duson and Carencro have come together to help 7-year-old Eli Prejean in his ongoing battle with a rare ...

A Q&A with Cormac Maher, MD, FAANS, FAAP, FACS; Division Chief, Pediatric Neurosurgery at Stanford Medicine Children’s Health ...

Human genes that encode proteins often contain non-coding segments known as introns. Removing introns is crucial for the proper expression of genetic information. Understanding how our cells ...

Gomekli is the second ever FDA-approved treatment for rare tumor disease, NF1-PN and the first to be approved for both adult and pediatric patients.

Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of ...

MRNA's competitive landscape, particularly against Pfizer, and higher forward P/S ratio suggest the stock is overvalued and ...

Recordati CEO Rob Koremans joined "Squawk Box Europe" after the pharmaceutical company raised its 2025 revenue and core profit forecasts by more than expected, seeing strong demand for its rare ...

A combination of human pedigree analysis and fruit fly genetics has identified a gene variant that appears to prevent PIGA-CDG, a rare but serious seizure disorder. The findings could ultimately lead ...

According to his parents, Eli is a “miracle baby,” surviving multiple hurdles since birth. Tune in TONIGHT at 5:00 p.m. to hear his story.

A young girl with a rare disease is preparing for a lengthy road to recovery. She’s hoping to decorate her hospital room with ...

Ascendis Pharma beat Q4 earnings and revenue estimates, driven by a $100M Novo Nordisk deal and Yorvipath's EU launch.