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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 patients with rare genetic mitochondrial diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted mavodelpar (REN001) Fast Track designation for long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency,…

Leriglitazone was well tolerated, and significant benefit was shown through clinically meaningful myelopathy endpoints and reduced progression of cerebral lesions Mataró, Barcelona, Spain, January 20, 2023 – Minoryx Therapeutics (“Minoryx”), a Phase 3 clinical stage biotech company focused on the development of differentiating treatment options in orphan central nervous system (CNS) disorders, today announces that data from its Phase 2/3…

FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that on January 10, 2023, the Compensation Committee of Mirum’s Board of Directors granted inducement awards consisting of non-qualified stock options to purchase 23,900 shares of common stock and 12,000 restricted stock units (“RSUs”) to four new employees under Mirum’s 2020 Inducement Plan. The Compensation…

SAN CARLOS, Calif., January 9, 2023 – Glycomine, Inc., a clinical-stage biotechnology company focused on developing new therapies for orphan diseases, today announced that the first patient has been dosed in a multi-center open-label Phase 2 study (ClinicalTrials.gov Identifier: NCT05549219) of GLM101, a targeted mannose-1-phosphate replacement therapy, in adult patients with phosphomannomutase 2-congenital disorder of glycosylation…

January 9, 2023 Download PDF EMERYVILLE, Calif., Jan. 09, 2023 (GLOBE NEWSWIRE) — 4D Molecular Therapeutics, Inc. (Nasdaq: FDMT), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, announced product pipeline portfolio updates and preclinical product candidate additions for its large market ophthalmology and pulmonology programs, as well as clinical…

– $76 million in total revenue and $74 million net product sales anticipated for LIVMARLI for 2022, preliminary and unaudited – sNDA for LIVMARLI in PFIC submission planned for first quarter 2023 – Multiple data analyses planned in 2023 advancing late-stage cholestasis programs FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today provided a preview…