Congenital, familial and genetic disorders – page 39

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 on Sotagliflozin for Improving Symptoms in Patients with Hypertrophic Cardiomyopathy

Study on Sotagliflozin for Improving Symptoms in Patients with Hypertrophic Cardiomyopathy

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

Study on Metoprolol for Preventing Heart Disease in Boys with Duchenne Muscular Dystrophy Aged 8-17

Study on Metoprolol for Preventing Heart Disease in Boys with Duchenne Muscular Dystrophy Aged 8-17

Study on the Effects of Metformin on Cognitive Function in Patients with Huntington's Disease

Study on the Effects of Metformin on Cognitive Function in Patients with Huntington's Disease

Study on Rigosertib Sodium for Patients with Recessive Dystrophic Epidermolysis Bullosa and Advanced Squamous Cell Carcinoma

Study on Rigosertib Sodium for Patients with Recessive Dystrophic Epidermolysis Bullosa and Advanced Squamous Cell Carcinoma

Study on the Effects of BT200 (Rondaptivon Pegol) for Patients with Type 2B von Willebrand Disease

Study on the Effects of BT200 (Rondaptivon Pegol) for Patients with Type 2B von Willebrand Disease

Study on Mesalamine to Prevent Colorectal Cancer in Patients with Lynch Syndrome

Study on Mesalamine to Prevent Colorectal Cancer in Patients with Lynch Syndrome