Congenital, familial and genetic disorders – page 58

Evaluating Higher Dose Nusinersen in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam

Study of venglustat tablets to reduce neuropathic and abdominal pain in patients aged 16 and older with Fabry disease who are untreated or haven't received treatment for 6 months

Study of venglustat tablets to reduce neuropathic and abdominal pain in patients aged 16 and older with Fabry disease who are untreated or haven't received treatment for 6 months

Study on the Effects of Leriglitazone in Male Children with Cerebral X-Linked Adrenoleukodystrophy Before Stem Cell Transplant

Study on the Effects of Leriglitazone in Male Children with Cerebral X-Linked Adrenoleukodystrophy Before Stem Cell Transplant

Long-Term Safety and Efficacy Study of Botaretigene Sparoparvovec for Patients with X-Linked Retinitis Pigmentosa

Long-Term Safety and Efficacy Study of Botaretigene Sparoparvovec for Patients with X-Linked Retinitis Pigmentosa

Study on Long-term Safety of VX-121, Tezacaftor, and Deutivacaftor in Patients with Cystic Fibrosis

Study on Long-term Safety of VX-121, Tezacaftor, and Deutivacaftor in Patients with Cystic Fibrosis

Study on R3R01 for Patients with Alport Syndrome and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

Study on R3R01 for Patients with Alport Syndrome and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

Study on Health, Activity, and Joint Outcomes in Patients Aged 13-69 with Severe or Moderate Hemophilia A Using Emicizumab

Study on Health, Activity, and Joint Outcomes in Patients Aged 13-69 with Severe or Moderate Hemophilia A Using Emicizumab

Long-term Safety and Efficacy Study of Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy

Long-term Safety and Efficacy Study of Onasemnogene Abeparvovec in Patients with Spinal Muscular Atrophy

Study on the Effectiveness of Decitabine and Tetrahydrouridine in Patients with Sickle Cell Disease Not Eligible for Hydroxyurea Treatment

Study on the Effectiveness of Decitabine and Tetrahydrouridine in Patients with Sickle Cell Disease Not Eligible for Hydroxyurea Treatment

Study of Nusinersen for Patients with Spinal Muscular Atrophy Previously Treated with Onasemnogene Abeparvovec

Study on NVD-003 Bone Graft for Treating Congenital Pseudarthrosis of the Tibia in Children

Study on NVD-003 Bone Graft for Treating Congenital Pseudarthrosis of the Tibia in Children

Study on Long-Term Safety and Efficacy of ARO-APOC3 for Adults with Mixed Dyslipidemia

Study on Long-Term Safety and Efficacy of ARO-APOC3 for Adults with Mixed Dyslipidemia