Rare Disease

August 6th 2025

Dordaviprone (Modeyso) is now approved to treat recurrent H3 K27M-mutant diffuse midline glioma, an ultra-rare, aggressive brain tumor primarily affecting children.

May 23rd 2025

Explore 5 rare genetic and neurological disorders named after pioneering women in medicine, highlighting their significant contributions to previously unknown conditions.

February 28th 2025

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, signifying progress in closing treatment gaps for rare disease.

February 12th 2025

Adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas that are not amenable to complete surgical resection are indicated in the approval.