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GLM101 is the first disease-modifying therapeutic in development to treat PMM2-CDG, the most common congenital disorder of glycosylation Data from the ongoing Phase 2 open-label study of GLM101 provide clinical proof of concept, showing improvement in ataxia, a key burden of disease in PMM2-CDG Funding will support advancement of GLM101 into a randomized, placebo-controlled Phase…

Jun 14, 2023 FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that data from LIVMARLI® (maralixibat) oral solution clinical studies evaluating patients with Alagille syndrome (ALGS) were published in Hepatology. LIVMARLI is the first and only approved treatment for cholestatic pruritus in patients with Alagille syndrome three months of age and older. The overall…

May 19, 2023 FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced data presented during the 55th European Society for Pediatric, Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual Meeting. Among the data presented were four oral and three poster presentations featuring analyses from LIVMARLI® (maralixibat) oral solution clinical studies. “We are thrilled to present data further…

AUGUST 30, 2023 SAN CARLOS, Calif., August 30, 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 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium taking place August 29 to September 1 in Jerusalem.…

Additional Phase 1 Data with CST-2032/CST-107 Reinforce Potential of Adrenoceptor Agonist Class of Drugs in Patients with Cognitive Impairment March 30, 2022 – Gothenburg, Sweden – CuraSen Therapeutics, Inc., a clinical-stage company developing small molecule drugs to treat neurodegenerative symptoms and disease progression, announced that it will present Phase 2 clinical data showing improvements in cognition…

CuraAX aims to address debilitating blood pressure drops and cognitive symptoms of nOH San Carlos, CA, – October 23, 2025 – CuraSen Therapeutics, Inc., a clinical-stage biopharmaceutical company developing drug candidates with broad applicability in neurodegenerative and neuropsychiatric diseases, today announced dosing of the first patient with CuraAX (CST-3056) in a Phase 2a proof-of-concept trial in…