Congenital, familial and genetic disorders – page 19

Study on Long-Term Safety of Luspatercept for Patients with Myelodysplastic Syndrome, Beta-Thalassemia, or Myelofibrosis Who Previously Participated in Luspatercept Trials

Study on Long-Term Safety of Luspatercept for Patients with Myelodysplastic Syndrome, Beta-Thalassemia, or Myelofibrosis Who Previously Participated in Luspatercept Trials

Study on Anakinra and Colchicine for Painful Attacks in Familial Mediterranean Fever Patients Resistant to Colchicine Therapy

Long-term Safety Study of Delandistrogene Moxeparvovec for Patients with Duchenne Muscular Dystrophy Who Previously Received SRP-9001

Long-term Safety Study of Delandistrogene Moxeparvovec for Patients with Duchenne Muscular Dystrophy Who Previously Received SRP-9001

Study on the Safety of VX-670 for Adults with Myotonic Dystrophy Type 1

Study on the Safety of VX-670 for Adults with Myotonic Dystrophy Type 1

Oxygen Therapy for Pregnant Women to Support Heart Development in Unborn Babies with Underdeveloped Left Heart Structures

Oxygen Therapy for Pregnant Women to Support Heart Development in Unborn Babies with Underdeveloped Left Heart Structures

Long-term Safety and Efficacy Study of JR-141 (Pabinafusp Alfa) for Male Patients with Hunter Syndrome (Mucopolysaccharidosis II)

Long-term Safety and Efficacy Study of JR-141 (Pabinafusp Alfa) for Male Patients with Hunter Syndrome (Mucopolysaccharidosis II)

Study on N-Acetyl-L-Leucine for Patients with Niemann-Pick Disease Type C

Study on Fazirsiran for Patients with Alpha-1 Antitrypsin Deficiency Liver Disease and Mild Fibrosis

Study on Fazirsiran for Patients with Alpha-1 Antitrypsin Deficiency Liver Disease and Mild Fibrosis

Study on Denosumab for Treating Fibrous Dysplasia/McCune-Albright Syndrome in Adults

Study on Denosumab for Treating Fibrous Dysplasia/McCune-Albright Syndrome in Adults

Study on the Safety and Tolerability of STK-002 for Patients with Autosomal Dominant Optic Atrophy

Study on the Safety and Tolerability of STK-002 for Patients with Autosomal Dominant Optic Atrophy

Continued Access Study for Patients with Severe Hemophilia A Using Efanesoctocog Alfa

Continued Access Study for Patients with Severe Hemophilia A Using Efanesoctocog Alfa

Study on the Safety and Effectiveness of TransCon CNP for Infants with Achondroplasia

Study on the Safety and Effectiveness of TransCon CNP for Infants with Achondroplasia