Rare Disease Day 2024 – Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy – special film launchedMarch 4, 2024Read article
Reneo Pharmaceuticals Announces Results from Pivotal STRIDE Study of Mavodelpar in Primary Mitochondrial Myopathies (PMM)February 5, 2024Read article
Glycomine Announces First Pediatric Patient Dosed in Phase 2 Clinical Study of GLM101, a Potential Treatment for PMM2-CDGFebruary 5, 2024Read article
Aura Biosciences Announces First Patient Dosed in Global Phase 3 CoMpass Trial Evaluating the Safety and Efficacy of Belzupacap Sarotalocan (Bel-sar) for First-Line Treatment of Early-Stage Choroidal MelanomaFebruary 5, 2024Read article
Minoryx announces enrollment of first patients with cerebral Adrenoleukodystrophy (cALD) in US Phase 3 clinical trial, CALYXDecember 13, 2023Read article
Glycomine Presents at ASHG 2023 Data from Ongoing Natural History Study with Insight into Genetic Mutations and Biomarkers for PMM2-CDGDecember 13, 2023Read article
Aura Biosciences Receives FDA Agreement Under Special Protocol Assessment (SPA) for CoMpass Phase 3 Clinical Trial of Belzupacap Sarotalocan (Bel-sar) in Early-stage Choroidal MelanomaDecember 13, 2023Read article
