Rare Disease

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.

November 15th 2024

Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve function after 52 weeks.

November 8th 2024

Perspectives of patients with spinal muscular atrophy (SMA) and their caregivers should be weighed before making decisions around SMA-enhancing treatments.