Rare Diseases Spinal Muscular Atrophy Inherited Diseases Hereditary Angioedema Mitochondrial Diseases Sanfilippo Syndrome Duchenne Muscular Dystrophy Blood Disorders Metabolic Disorders CPS1 Deficiency Sickle Cell Disease Urea Cycle Disorders Hereditary Conditions Epidermolysis Bullosa Cystic Fibrosis Gene Therapy Creatine Transporter Deficiency Hereditary Cancer Syndromes Gene Editing Inherited Conditions Cardiovascular Diseases Diagnosis Anhidrosis Methylmalonic Acidemia Symptoms and Treatment Diagnostic Methods Fibrodysplasia Ossificans Progressiva Treatment Options Beta-Thalassemia Neurodegenerative Diseases Noonan Syndrome Familial Chylomicronemia Syndrome Osteogenesis Imperfecta Nonsense Mutations Patient Care SMA Cardiac Conditions Hypertrophic Cardiomyopathy Cardiac Health Mucopolysaccharidosis Menkes Disease Osteopetrosis Thalassaemia Niemann-Pick Disease Hypercholesterolemia Immunodeficiency Disorders Amyotrophic Lateral Sclerosis Trisomy 13 Adenosine Deaminase Deficiency Developmental Disorders Severe Immunodeficiencies Fanconi Anemia Genetics Angelman Syndrome Familial Hypercholesterolemia Vitamin A Metabolism Muscular Dystrophy Hemoglobinopathies Congenital Hypogonadotropic Hypogonadism Severe Combined Immunodeficiency
Early use cut mortality by nearly 80%, offering a targeted option for a fatal copper-transport disorder.
