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Mitochondrial Disease News


Rare Disease Day at NIH, Set for March 1, Growing Year by Year

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...


What is Leigh Syndrome? If you had MS, I could help you

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,...


Ultragenyx Announces Approval of Dojolvi™ (UX007/triheptanoin) in Canada for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Adults and Children

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...


‘I put my life on hold:’ Disability groups plead for vaccine

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...


Mile-wide smile as Abby MacCurtain of Plymouth celebrates 10th birthday

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...


Mitochondrial-based Therapies Offer New Ways to Tackle Acute and Chronic Conditions

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...


Financial Advocacy in RARE: Navigating the U.S. Health System for Young Adults

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...


Working to Treat Mitochondrial Epilepsy – One Seizure at a Time

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...


Medical Pioneer Fought Relentlessly for Patients’ Lives

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...


Pediatric mitochondrial disease and COVID-19 FAQ

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...