An Existing Drug May Have Therapeutic Potential in Mitochondrial Disease --CHOP Researchers: Animal Studies May Offer Clues to Possible Precision Medicine Approach-- Philadelphia, Feb. 26, 2019 --New preclinical findings from extensive cell and animal studies suggest that a drug already used for a rare kidney disease could benefit patients with some mitochondrial disorders—complex [...]
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So far Cliff Gorski has created 78 blog entries.
An interdisciplinary research team has been awarded $395,112 to develop new tools to assess mitochondrial diseases, which are difficult to diagnose and treat, and can be fatal in children and adults. The National Institutes of Health have awarded researchers from the College of Engineering and the J. William Fulbright College of Arts and Sciences [...]
UMDF supports The National Organization for Rare Disorders (NORD)Ⓡ, in their new campaign for Rare Disease Day centering on three simple words: Show Your Stripes. The campaign is a call to action for people to literally and figuratively “show their stripes” in support of rare diseases leading up to and on Rare Disease Day, [...]
Patients with mitochondrial disease have a significantly higher cost burden compared with the general population, according to the results of a recent retrospective claims analysis. See the report here.
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Baltimore, MD , December 19, 2018 -- The Johns Hopkins University School of Medicine and Kennedy Krieger Institute have jointly been named a certified Mitochondrial Medicine Center (MMC). Mitochondrial disease affects tiny structures within the cell, called mitochondria, that are often called the "powerhouses" of the cell. The disease is inherited, and it causes [...]
(Pittsburgh, PA) -- The Foundation for Mitochondrial Medicine (FMM) and the United Mitochondrial Disease Foundation (UMDF) are excited to announce an agreement to combine and advance their synergistic missions of advancing research, education and patient support for treatments of mitochondrial disease. FMM made this decision after careful strategic consideration for opportunities to generate greater [...]
Stealth BioTherapeutics Granted Fast Track Designation for Elamipretide for the Treatment of Dry Age-Related Macular Degeneration with Geographic AtrophyCliff Gorski2018-12-10T12:00:13+00:00
BOSTON, Dec. 10, 2018 /PRNewswire/ -- Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its lead investigational candidate, elamipretide, for the treatment of dry age-related macular degeneration (AMD) with geographic atrophy. "We are pleased the FDA [...]
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The following is a summary of a live webinar presented by Reenie McCarthy, CEO and Jim Carr, PharmD, Chief Clinical Development Officer of Stealth BioTherapeutics (Stealth BT). All investigational product candidates mentioned in this summary are investigational new drugs. They are not approved by the FDA for the treatment of any disease, condition or [...]