Danish pharmaceutical multinational Novo Nordisk is expanding its presence in inherited blood disorder treatments ...

BioMarin is acquiring Amicus Therapeutics for $4.8 billion, merging two rare disease-focused biotechs founded around the turn ...

Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes ...

U.S. health officials have added two deadly rare diseases to the list of conditions the government recommends states screen ...

Ipsen’s oral small molecule has failed to reduce the progression of a genetic disease that gradually turns tendons and ...

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...

U.S. Health Secretary Robert F. Kennedy Jr. on Tuesday added two rare genetic disorders, Duchenne Muscular Dystrophy and ...

Lee meets with rare disease-stricken patients President Lee Jae Myung (rear, 5th from L), alongside his wife, Kim Hea Kyung ...

Health secretary Robert F. Kennedy Jr. on Tuesday added two deadly rare diseases, Duchenne muscular dystrophy and ...

The donation from Don and Anne Edwards aims to speed research, treatment and diagnosis for children with rare and genetic ...

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...

Addition joins a growing list of launches this year, following in the footsteps of startups like Crystalys Therapeutics and ...