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EXPERTS-ALS: EXPErimental medicine Route To Success in Amyotrophic Lateral Sclerosis
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EMBARC-AIRNET: The EMBARC Anti-Inflammatory Repurposing Network
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FARGO: FAecal Microbiota Transplantation in primaRy sclerosinG chOlangitis
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STOPFOP: Saracatinib Trial TO Prevent FOP
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Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type II
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PKAN: Phase II Study of a Novel Vitamin Metabolite (CoA-Z) for Pantothenate Kinase Associated Neurodegeneration
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DefINe: A trial to test the use of deferiprone in people with neuroferritinopathy
