The Food and Drug Administration (FDA) has granted Orphan Drug designation to REN001 (Reneo Pharmaceuticals) for the treatment of fatty acid oxidation disorders. REN001 is a selective peroxisome ...
Fatty acid oxidation (FAO) disorders comprise a heterogeneous group of inborn errors of metabolism that impair the mitochondrial capacity to convert fatty acids into energy. These inherited conditions ...
Credit: FDA. A New Drug Application (NDA) has been submitted to the Food and Drug Administration (FDA) for triheptanoin (Ultragenyx) for the treatment of long-chain fatty acid oxidation disorders. A ...
Additional 20 Patients Naïve to UX007 Also Demonstrated Meaningful Reductions in Major Clinical Events with Treatment Company On Track for mid-2019 NDA Submission NOVATO, Calif., Jan. 22, 2019 (GLOBE ...
IRVINE, Calif., Jan. 31, 2023 (GLOBE NEWSWIRE) -- Reneo Pharmaceuticals, Inc. (Nasdaq: RPHM), a clinical-stage pharmaceutical company focused on the development and commercialization of therapies for ...
NOVATO, Calif., July 22, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare ...
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