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Mitochondrial Disease News


Prominent Mitochondrial Researcher Dr. Navdeep Chandel Joins Penrose TherapeuTx Scientific Advisory Board

Wed, March 10, 2021, 7:00 AM·3 min read Dr. Chandel brings decades of research expertise as the company advances its novel Mitochondria Modifying Agent oncology research program Penrose TherapeuTx,...


Researchers find effective combination of therapies for managing mitochondrial disease

Study is first to demonstrate how one specific combinational therapy may provide measurable benefits to patients with mitochondrial respiratory chain disorders, which currently lack FDA-approved therapies CHILDREN’S HOSPITAL OF...


Having a Rare Disease Is a Lot Harder Than You Think

Because we just honored Rare Disease Day on Feb. 28, I’d like to share what it’s like living with one. Having a rare disease means strangers telling you what your rare disease means,...


Welcome to Rare Disease Day: An Interview with a NORD Representative

Lisa Sarfaty, director of strategic planning for the National Organization for Rare Disorders, speaks on the role of the organization and the importance of the Rare Disease Day campaign....


Expediting innovation in the rare disease domain

Rare diseases affect a fraction of the population – 450 million+ people globally. However, its effects can be equally severe and life-threatening as other prevalent diseases. For Rare Disease Day,...


4 Reasons to Include Coq10 in Your Diet

Coenzyme Q10, or Coq10, is known in the health industry as a powerful antioxidant and plays an important role in metabolism. It is a compound that the body produces...


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...