Dec 16 (Reuters) - U.S. Health ‌Secretary ​Robert F. Kennedy ‌Jr. on Tuesday added two rare ​genetic disorders, Duchenne ...

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

The nonprofit is funding research at two universities—Oregon Health & Science University and UMass Chan Medical School—on a ...

As the FDA unveils a parade of initiatives aimed at accelerating drug development for rare diseases, experts appeal for a ...

Gene editing has the potential to cure thousands of people suffering from devastating rare diseases. But the Nobel ...

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

Ohio State student Cailin Joyce died after suffering a rare blood disorder caused by the Epstein-Barr virus that “left her ...

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

The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...

Soleo Health has appointed Jody Thompson as senior vice president of business development, rare disease, to broaden the company’s portfolio of exclusive and limited distribution therapies for rare, ...

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 ...

Juvenile Huntington’s disease is more aggressive than Huntington’s disease, reducing a child’s life expectancy at a far ...