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…

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…

NOVEMBER 2, 2023 SAN CARLOS, Calif., November 3, 2023 – Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, announced the presentation of summary findings from its ongoing natural history study at the American Society for Human Genetics (ASHG) 2023 Annual Meeting taking place in Washington, DC.  The findings included genotype characterization, combined with…

OCTOBER 12, 2023 SAN CARLOS, Calif., October 12, 2023 – Glycomine, Inc., a clinical-stage biotechnology company focused on developing new therapies for serious orphan diseases, announced the appointment of Rose Marino, M.D. as Chief Medical Officer. Dr. Marino has more than 20 years of collective experience in academic medicine and clinical development in the pharmaceutical industry,…

DECEMBER 11, 2023 SAN CARLOS, Calif., December 11, 2023 – Glycomine, Inc., a clinical-stage biotechnology company focused on developing new therapies for orphan diseases, announced the first pediatric patient has been dosed in a multi-center open-label Phase 2 study of GLM101, a mannose-1-phosphate replacement therapy, in patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG). “The initiation of dosing…