Launched on YouTube, Rare Connections in NMOSD seeks to raise awareness of the film through testimonials of three patients living with the rare inflammatory condition.
Healthcare brands, agencies and patients recognize Rare Disease Day 2024
In New York, the Empire State Building will be illuminated in a show of support, as will the Nasdaq MarketSite in Times Square and building 1 at the Food and Drug Administration headquarters in Jersey City, New Jersey.
Amgen details rare disease ambitions following $28B Horizon acquisition
“Most Likely Not To” will premiere at the Times Center in New York on February 29 — which is Rare Disease Day — and stars members of the SMA community, who also wrote the show.
Novartis unveils promising Fabhalta data for C3 glomerulopathy indication
Topline results from the Phase 3 trial, APPEAR-C3G, showed Fabhalta (iptacopan) met its primary endpoint in reducing protein in the urine in patients with C3G.
FDA adopts Operation Warp Speed lessons for rare disease pilot program
The FDA announced the launch of a pilot program, dubbed START, to address challenges associated with rare disease development and speed up the regulatory process.
Queer Eye designer sets sights on rare blood cancer in GSK effort
Given Jimmy Buffett’s prominence in popular culture and sudden death due to a rare form of skin cancer, it’s worth exploring what Merkel cell carcinoma is.
A move to cut drug prices has patients with rare diseases worried