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Glycomine Presents at ASHG 2023 Data from Ongoing Natural History Study with Insight into Genetic Mutations and Biomarkers for PMM2-CDG

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…

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Glycomine Receives FDA Fast Track Designation for GLM101 for the Treatment of PMM2-CDG

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…

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Hepatology Communications Publishes Data from Mirum’s LIVMARLI in Primary Sclerosing Cholangitis

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…

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Leriglitazone has met the primary endpoint in NEXUS, the pivotal trial for pediatric patients with cerebral Adrenoleukodystrophy

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…

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