Congenital, familial and genetic disorders – page 38

Study on NTLA-2002 for Treating Hereditary Angioedema in Patients: Evaluating the Effects of Ziclumeran and Lonvoguran

Study on NTLA-2002 for Treating Hereditary Angioedema in Patients: Evaluating the Effects of Ziclumeran and Lonvoguran

Study of Deucrictibant Extended-Release Tablets for Prevention of Hereditary Angioedema Attacks in Adults and Adolescents

Study of Deucrictibant Extended-Release Tablets for Prevention of Hereditary Angioedema Attacks in Adults and Adolescents

Study of Ruxolitinib with Brentuximab or Pembrolizumab for Patients with Relapsed or Refractory Classical Hodgkin Lymphoma

Study of Ruxolitinib with Brentuximab or Pembrolizumab for Patients with Relapsed or Refractory Classical Hodgkin Lymphoma

Title: Safety and effectiveness study of pegzilarginase weekly injections in children under 2 years old with Arginase 1 Deficiency

Title: Safety and effectiveness study of pegzilarginase weekly injections in children under 2 years old with Arginase 1 Deficiency

Long-Term Study on Oxytocin for Children with Prader-Willi Syndrome Up to Age 4

Long-Term Study on Oxytocin for Children with Prader-Willi Syndrome Up to Age 4

Study on the Effects and Safety of JR-141 and Idursulfase for Patients with Hunter Syndrome

Study on the Effects and Safety of JR-141 and Idursulfase for Patients with Hunter Syndrome

Study on the Effects of GTX-102 for Children with Angelman Syndrome

Study on the Effects of GTX-102 for Children with Angelman Syndrome

Study of Vosoritide for Children with Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome Not Responding to Growth Hormone

Study of Vosoritide for Children with Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome Not Responding to Growth Hormone

Study on the Effectiveness and Safety of Venglustat for Adults and Children with Gaucher Disease Type 3

Study on the Effectiveness and Safety of Venglustat for Adults and Children with Gaucher Disease Type 3

Study on Pramipexole for Treating Restless Legs in Women with X-linked Adrenoleukodystrophy

Study on Pramipexole for Treating Restless Legs in Women with X-linked Adrenoleukodystrophy

Study on the Safety and Effects of Saracatinib for Patients with Fibrodysplasia Ossificans Progressiva (FOP)

Study on the Safety and Effects of Saracatinib for Patients with Fibrodysplasia Ossificans Progressiva (FOP)

Study on Higher Doses of Nusinersen for Patients with Spinal Muscular Atrophy Who Previously Participated in a Nusinersen Study