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.
IFFGD Will Participate in Rare Disease Day to Support Rare Digestive Disease Patients
The International Foundation for Functional Gastrointestinal Disorders will participate in Rare Disease Day on February 28, with efforts to support patients affected by a rare digestive disease globally.
Alzheimer's Treatment Prevents and Improves Neurological Damage on Mouse Model
A collaborative research effort between the National Institute on Aging (NIA), the National Institutes of Health (NIH), and ChromaDex has found Niagen effective in preventing neurological damage and improving cognitive and physical function in a mouse model of Alzheimer’s disease.
Amplify Hope Study Educates Families on Crowdfunding for Genetic Testing
The Amplify Hope Study, sponsored by the Rare Genomics Institute, analyzed the effects of educating families affected by rare disease on how to effectively crowdfund to pay for necessary genetic testing.
CHLA Takes On Trial of Potential Stem Cell Procedure in Treating HLHS
Children’s Hospital Los Angeles (CHLA) has announced that it will be participating in a new clinical trial with the hopes of improving quality of life (QOL) outcomes for children with hypoplastic left heart syndrome (HLHS), a congenital heart defect in which the left ventricle is severely underdeveloped.
BioPontis Alliance and Massachusetts General Hospital Partner for HD
February 2nd 2018Yesterday, nonprofit organization BioPontis Alliance and Massachusetts General Hospital, the largest hospital-based research program in the United States, announced an agreement to target new treatments for Huntington’s disease.