News

2023-11-16 As part of CALYX, Minoryx is conducting an extensive pre-screening MRI-based campaign aimed at identifying eligible adult X-ALD patients with cerebral Adrenoleukodystrophy (cALD) Mataró, Barcelona, Spain, November 16, 2023 – Minoryx Therapeutics, a registration stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces the first patients have…

 4 March 2025 Barcelona, Spain – 4 March 2025 – Minoryx Therapeutics, a late-stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces that the first patient in the TREE study has been dosed with leriglitazone. TREE is a phase 2a clinical study assessing the safety and efficacy…

2024-12-11 Statistically significant difference in arrested disease in leriglitazone treated patients when compared to the natural history of the disease Submission of European marketing authorization application (MAA) for leriglitazone expected mid-2025. Barcelona, Spain and Düsseldorf, Germany – 11 December 2024 – Minoryx Therapeutics, a late-stage biotech company focused on the development of therapies for orphan central…

Apr 06, 2023 FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that Tim Walbert has been appointed to the company’s Board of Directors. In addition to Mr. Walbert’s appointment, the company shared that Niall O’Donnell, Ph.D., a Mirum director since November 2018, has announced his plan to step down from the Board of…

May 18, 2023 FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that Hepatology Communications published data from the CAMEO study evaluating the safety, tolerability, and efficacy of LIVMARLI® (maralixibat) oral solution in patients with primary sclerosing cholangitis (PSC). The CAMEO study was the first proof-of-concept study evaluating an ileal bile acid transporter (IBAT) inhibitor in…

September 18, 2024 SAN CARLOS, Calif., September 18, 2024 – Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for GLM101, a mannose-1-phosphate replacement therapy in development for the treatment of patients with phosphomannomutase 2-congenital disorder…