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C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia.
A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.
An Anti-CD3 Antibody, Teplizumab, in Relatives at Risk for Type 1 Diabetes.
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
AR101 Oral Immunotherapy for Peanut Allergy.
Trial of Fingolimod versus Interferon Beta-1a in Pediatric Multiple Sclerosis.
A Phase 3 Trial of l-Glutamine in Sickle Cell Disease.
Sodium Thiosulfate for Protection from Cisplatin-Induced Hearing Loss.
Burosumab Therapy in Children with X-Linked Hypophosphatemia.
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.