Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a...
Orphanet Journal of Rare Diseases is proud to present this piece, a runner-up entry in Findacure and Medics4RareDiseases’ “Student Voice” contest, which explores some of the changes which need to be made in healthcare,...
February 17, 2021 08:30 ET | Source: Ultragenyx Pharmaceutical Inc. NOVATO, Calif., Feb. 17, 2021 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization...
Though many people with disabilities are more vulnerable to COVID-19, in some U.S. states they’re being left behind in a massive effort to get limited vaccines into the arms...
PLYMOUTH — Out on a routine call, the fire engine moves slowly quietly though the neighborhood. Bundled up against a blustery breeze in her wheelchair, Abby MacCurtain spots it as is...
Published: Feb 12, 2021 By Gail Dutton Photo Courtesy of CohBar, Inc. CohBar, Inc. is pioneering mitochondrial-based therapies with a candidate in a Phase Ia/Ib clinical trial as a potential treatment...
The transition to adulthood is a process and the experience of becoming an adult is different for everyone whether or not you have a rare disease. But if you...
FEBRUARY 8, 2021 Robert F. Kennedy famously said, “The purpose of life is to contribute in some way to making things better.” And when you’re in the rare disease...
American medicine lost a giant this week. His passing, though, reminds us that no matter how great the medical challenge — cancer, heart disease, diabetes, polio, neurodegenerative diseases, the Spanish...
What is pediatric mitochondrial disease? How does the COVID-19 pandemic affect children with this disease? Click here to read the full article written by Dr. Peter McGuire at the...