The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.
April 29th 2024
FDA approves mavorixafor (XOLREMDI) for WHIM syndrome, the first approved treatment for the rare disease.
New Data Suggests Changes to Existing Huntington's Disease Treatment Approach
February 1st 2018Rockefeller University scientists have observed the effects of Huntington’s disease in neurons as early as conception, and suggest therapies that block HTT protein activity may be doing more harm than good.
FDA Allows Fibrocell to Initiate Pediatric Enrollment in Epidermolysis Bullosa Trial
January 30th 2018This morning, Fibrocell Science, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted the company allowance to begin enrollment in the Phase 2 portion of its Phase 1/2 clinical trial of FCX-007.
FIREFISH Study Shows Clinical Benefit of RG7916 in Infants With SMA
PTC Therapeutics has announced that Part 1 of the FIREFISH study has shown that RG7916, a Type 1 spinal muscular atrophy (SMA) drug, to be safe and well-tolerated in infants and babies with Type 1 SMA.
Financial Coverage for Rare Disease Treatments Inconsistent in British Columbia
The Canadian Agency for Drugs and Technologies in Health organization’s Common Drug Review protocol often leaves rare disease patients in British Columbia without governmental payment coverage for their expensive treatments.