BioMarin, Jazz Pharmaceuticals, and Otsuka CEOs to Join NORD CEO to Examine What it Takes to Turn Scientific Advancements ...

After being diagnosed with Alagille syndrome, a rare disease, at nearly two months old, three-year-old N.M. and the child’s family have gradually become accustomed to frequent hospital visits in ...

The first-ever international guidelines on managing childhood obesity are raising the profile of GLP-1 treatments for kids ...

More than 1 million people in the United States are currently living with Parkinson's disease. Everyone's story is unique and ...

People with rare diseases are being offered two years of free AI app access to capture medical appointments and summarise ...

A Tiffin, IA biotech company is building a biological resource that could help researchers better understand rare diseases and pursue future diagnostics and treatments.

CureSPG50 and SickKids to pursue Phase 1/2 clinical trial of potential gene therapy for CLN7 Batten disease in Canada ...

ATSN-201 demonstrated structural and functional benefit in Parts A and B, including microperimetry improvements exceeding the level the FDA has ...

Dermatologists say biotin supplements and shampoos are unlikely to help with hair loss. Here is what does.

Solengepras 150 mg achieved a statistically significant reduction in daily OFF time, with benefit seen at Week 2Statistically ...

AMO Pharma Limited ("AMO Pharma"), a privately held clinical-stage specialty biopharmaceutical company focusing on rare genetic disorders with limited or no treatment options, in collaboration with ...

A new case report from Yemen describes an extremely rare coexistence of necrotizing fasciitis and myiasis in a 38-year-old ...