News

Increased Education Crucial to Improving Rare Disease Care, Survey Finds

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…

New Rules in UK Allow More Black Donors to Give Blood for SCD

Blood donation restrictions in England that had prevented at least 12,000 Black donors from giving blood were lifted by the country’s government late last year in what the Sickle Cell Society called “a landmark change.” Now, citing “an urgent need” in the U.K. for blood donation from Black donors…

GBT Foundation Invites SCD Care Access Programs to Try for Grants

The GBT Foundation is calling for proposals for its Access to Care Empowerment for Sickle Cell (ACE) grant program, which will provide up to $250,000 to community-based sickle cell disease (SCD) organizations to accelerate development of care access programs for SCD patients. The ACE program, which was established this…

#RAREis Representation Program Promotes Equity, Diversity

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…

FDA Grants Orphan Drug Status to Naproxcinod for Pain Crises

Naproxcinod, an investigational therapy being developed by Fera Pharmaceuticals as a treatment for sickle cell disease (SCD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). Orphan drug status provides regulatory support and financial benefits to experimental treatments for diseases that affect fewer…