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Mirum Submits for European Approval of LIVMARLI in Progressive Familial Intrahepatic Cholestasis

Apr 05, 2023 FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced submission to the European Medicines Agency (EMA) of a variation application to the Marketing Authorization for LIVMARLI® (maralixibat) oral solution, to extend the label for progressive familial intrahepatic cholestasis (PFIC) in patients two months of age and older. LIVMARLI is currently approved by…

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Mirum’s LIVMARLI® Now Authorized in Canada for Cholestatic Pruritus in Patients with Alagille Syndrome

Jul 25, 2023 FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that LIVMARLI® (maralixibat oral solution) has been authorized by Health Canada for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20230725391772/en/ LIVMARLI (maralixibat oral solution) (Graphic: Business Wire) ALGS…

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Multiple Clinical Complete Responses Demonstrated Following Single Low Dose Administration of Bel-sar in Patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) in Ongoing Phase 1 Trial

October 17, 2024 Clinical Complete Responses Observed in 4 out of 5 Patients in Subset of Patients with Low Grade Disease; Evidence of Bladder Urothelial Field Effect in Non-Target Tumors Favorable Safety Profile Observed; Only Grade 1 Drug-Related Adverse Events Reported in Less Than 10% of Patients Aura Hosting Virtual Urologic Oncology Investor Event with Key Opinion…

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Rare Disease Day 2024 – Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy – special film launched

2024-02-29 Rare Disease Day 2024 – Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy – special film launched Barcelona, Spain and Düsseldorf, Germany – 29 February, 2024 – Neuraxpharm Group (Neuraxpharm), a leading European specialty pharmaceutical company focused on the treatment of central nervous system (CNS) disorders, and Minoryx Therapeutics, a late stage biotech…

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Reneo Announces Pricing of Public Offering of Common Stock

05/03/2023 IRVINE, Calif., May 03, 2023 (GLOBE NEWSWIRE) — Reneo Pharmaceuticals, Inc. (Reneo) (Nasdaq: RPHM), a clinical-stage pharmaceutical company focused on the development and commercialization of therapies for patients with rare genetic mitochondrial diseases, today announced the pricing of its previously announced underwritten public offering of 6,875,000 shares of its common stock at a public…

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Reneo Pharmaceuticals Achieves Target Enrollment in the Pivotal STRIDE Study of Mavodelpar (REN001) in Primary Mitochondrial Myopathies

Topline data from the STRIDE study expected in the fourth quarter of 2023 IRVINE, Calif., March 14, 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 company has achieved the target enrollment…

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