Congenital, familial and genetic disorders – page 14

Study of Piperacillin/Tazobactam and Levofloxacin for Patients with COPD or Cystic Fibrosis

Study of Piperacillin/Tazobactam and Levofloxacin for Patients with COPD or Cystic Fibrosis

Study of Aficamten for Children with Symptomatic Obstructive Hypertrophic Cardiomyopathy

Study of Aficamten for Children with Symptomatic Obstructive Hypertrophic Cardiomyopathy

Study of Nipocalimab or IVIG for Pregnant Women at Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)

Study of Nipocalimab or IVIG for Pregnant Women at Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)

Study on the Safety and Effectiveness of BEAM-302 with MR0005 and GR0015 for Adults with Alpha-1 Antitrypsin Deficiency-Related Lung or Liver Disease

Study on the Safety and Effectiveness of BEAM-302 with MR0005 and GR0015 for Adults with Alpha-1 Antitrypsin Deficiency-Related Lung or Liver Disease

Study on the Safety and Effectiveness of CTX001 for Children with Severe Sickle Cell Disease Using Exagamglogene Autotemcel, Busulfan, and Plerixafor

Study on the Safety and Effectiveness of CTX001 for Children with Severe Sickle Cell Disease Using Exagamglogene Autotemcel, Busulfan, and Plerixafor

Study on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination

Study on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination

Study on the Effectiveness of Ambroxol and Clenbuterol in Children and Adults with Gaucher Disease TYPE 3

Long-Term Safety Study of Berotralstat for Patients with Hereditary Angioedema from Previous Trials

Long-Term Safety Study of Berotralstat for Patients with Hereditary Angioedema from Previous Trials

Study of GNT0003 and imlifidase in adults with Crigler-Najjar syndrome who require daily phototherapy and have pre-existing AAV8 antibodies

Study of GNT0003 and imlifidase in adults with Crigler-Najjar syndrome who require daily phototherapy and have pre-existing AAV8 antibodies

Study on the Effectiveness of Apixaban for Pain Relief in Patients with Venous Malformations

Study on the Effectiveness of Apixaban for Pain Relief in Patients with Venous Malformations

Long-Term Safety Study of Gene Therapy with Etuvetidigene Autotemcel for Patients with Wiskott-Aldrich Syndrome

Long-Term Safety Study of Gene Therapy with Etuvetidigene Autotemcel for Patients with Wiskott-Aldrich Syndrome

Study of Dostarlimab Combined with Short Course Radiotherapy for Locally Advanced Rectal Cancer Patients with Microsatellite Instability

Study of Dostarlimab Combined with Short Course Radiotherapy for Locally Advanced Rectal Cancer Patients with Microsatellite Instability