Congenital, familial and genetic disorders – page 27

Study of the Safety, Tolerability and Efficacy of Oral Vafidemstat in Adults with Phelan‑McDermid Syndrome (HOPE‑2)

A study of etavopivat to reduce vaso‑occlusive crises in children with sickle cell disease

A study of etavopivat to reduce vaso‑occlusive crises in children with sickle cell disease

Study of aficamten and 68Ga‑FAPI‑46 versus placebo in asymptomatic obstructive hypertrophic cardiomyopathy patients

Study of aficamten and 68Ga‑FAPI‑46 versus placebo in asymptomatic obstructive hypertrophic cardiomyopathy patients

Levofloxacin prophylaxis to reduce bloodstream infections in patients with hematologic malignancies undergoing allogeneic hematopoietic stem cell transplantation

Levofloxacin prophylaxis to reduce bloodstream infections in patients with hematologic malignancies undergoing allogeneic hematopoietic stem cell transplantation

Efficacy and safety of oral venglustat versus intravenous imiglucerase in children aged 2‑11 years with Gaucher disease type 3

Efficacy and safety of oral venglustat versus intravenous imiglucerase in children aged 2‑11 years with Gaucher disease type 3

Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients with Gaucher Disease Type 1 Using Drug Combination

Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients with Gaucher Disease Type 1 Using Drug Combination

Safety and Efficacy of Intrathecal BIIB115 in Infants with Spinal Muscular Atrophy Previously Treated with Onasemnogene Abeparvovec

Safety and Efficacy of Intrathecal BIIB115 in Infants with Spinal Muscular Atrophy Previously Treated with Onasemnogene Abeparvovec

Efficacy and Safety of Lucerastat in Treatment‑Naïve Adult Male Patients with Fabry Disease

Efficacy and Safety of Lucerastat in Treatment‑Naïve Adult Male Patients with Fabry Disease

Phase 3 study of mitapivat for effectiveness and safety in children with non‑transfusion‑dependent alpha or beta thalassemia

Phase 3 study of mitapivat for effectiveness and safety in children with non‑transfusion‑dependent alpha or beta thalassemia

Surgery versus Lauromacrogol 400 injection for superficial labial venous malformations in children and adults: a randomized controlled trial

Surgery versus Lauromacrogol 400 injection for superficial labial venous malformations in children and adults: a randomized controlled trial

Phase 3 Study of Gildeuretinol Acetate (ALK‑001) to Assess Safety and Efficacy in Slowing Progression of Stargardt Disease in Adults

Phase 3 Study of Gildeuretinol Acetate (ALK‑001) to Assess Safety and Efficacy in Slowing Progression of Stargardt Disease in Adults

Phase 3 Study of Mitapivat to Reduce Transfusions in Children with Alpha or Beta Transfusion‑Dependent Thalassemia

Phase 3 Study of Mitapivat to Reduce Transfusions in Children with Alpha or Beta Transfusion‑Dependent Thalassemia