Primary safety, pharmacokinetics and secondary efficacy endpoints met, with consistent improvements in RSBQ Total scores and CGI-I Efficacy endpoints demonstrated statistically significant and clinically meaningful reductions in
(COE) at Children’s Hospital of Philadelphia (CHOP) was awarded $1.275 million by the Friedreich’s Read More
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...
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...
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%...
“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
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,...
MITOCHONDRIAL DISEASE TRIAL: Dec 1, 2020 15:30 UTC TOKYO–(Business Wire)–LUCA Science (Tokyo, Japan), a biotechnology company pioneering a novel...
cellvie , a company based in Houston, Texas, is developing a mitochondrial transplantation therapy to help reduce the damage...