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Mitochondrial Disease News


Pennington Biomedical to select 375 participants for 2021 research study regarding benefits of exercise

Engaging in regular exercise is typically viewed as a positive activity that results in improved physical and mental health. But, does regular exercise affect some people more positively than...


What does space do to the human body? 29 studies investigate the effects of exploration

INCLUDES EFFECTS ON MITOCHONDRIA: JAXA astronaut Akihiko Hoshide finishes a blood collection as part of Expedition 32 in 2012. With biological data from a whopping 56 astronauts, scientists have...


Larimar Therapeutics Announces Completion of Dosing of the Single Ascending Dose Clinical Trial in Friedreich’s Ataxia Patients and Provides Program Update

RE: FRIEDRICH’S ATAXIA: – Preliminary data suggest that single subcutaneous injections of CTI-1601 were well tolerated at doses up to 100 mg – Company remains on track to report...


Poxel Announces Additional Positive Phase 2a Results, and Phase 2b Plan for PXL770, an Oral First-in-Class AMPK Activator, in NASH

NASH/DIABETES 2 RELATED: New results from the Phase 2a STAMP-NAFLD trial show consistently greater response in high-risk patients with coexisting type 2 diabetes (T2DM), estimated to be approximately 50%...


Family’s tribute to ‘handsome and happy’ Charlie, 6, who died from mitochondrial disease

“Always smiling” Charlie Nicholls, aged six, was an inspiration to many people. He tragically died six weeks ago from gut failure, which was secondary to his mitochondrial disease. Picture:...


Sarepta Therapeutics Announces Positive Clinical Results from MOMENTUM, a Phase 2 Clinical Trial of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51

RE: DUCHENNES: Results from the multiple-ascending dose trial demonstrate proof-of-concept for SRP-5051 and support continued dose escalation — — At a total dose exposure approximately 10x lower than eteplirsen,...


LUCA Science Announces Joint Research With Division of Cardiology, Field of Internal Medicine, Nagoya University Graduate School of Medicine on the Development of Mitochondrial Therapy for Myocardial Infarction (Cardiac Ischemic-reperfusion Injury)

MITOCHONDRIAL DISEASE TRIAL: Dec 1, 2020 15:30 UTC TOKYO–(Business Wire)–LUCA Science (Tokyo, Japan), a biotechnology company pioneering a novel class of mitochondrial therapeutics, announced today that it will collaborate...


Mitochondrial Transplantation to Treat Ischemia-Reperfusion Injury: Interview with Dr. Alexander Schueller, CEO of cellvie

cellvie , a company based in Houston, Texas, is developing a mitochondrial transplantation therapy to help reduce the damage caused by ischemia-reperfusion injury. The treatment involves delivering isolated mitochondria...


Larimar Therapeutics Announces Completion of Dosing of the Single Ascending Dose Clinical Trial in Friedreich’s Ataxia Patients and Provides Program Update

RE: FRIEDRICH’s ATAXIA- Preliminary data suggest that single subcutaneous injections of CTI-1601 were well tolerated at doses up to 100 mg – Company remains on track to report topline...


PTC Announces Translarna™ Approval in Russia for the Treatment of Duchenne Muscular Dystrophy

SOUTH PLAINFIELD, N.J., Dec. 4, 2020 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Translarna™ (ataluren) has been granted marketing approval in the Russian Federation for nonsense...