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EXPErimental medicine Route To Success in Amyotrophic Lateral Sclerosis (EXPERTS-ALS)
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AIR-NET: Testing anti-inflammatories for the treatment of bronchiectasis
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FARGO: FAecal Microbiota Transplantation in primaRy sclerosinG chOlangitis
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TIARA: Autologous Tregs for Aplastic Anaemia
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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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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
