Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

Medicine and Dentistry

• Patient 100%
• Clinical Research 60%
• Age 40%
• Werdnig Hoffmann Disease 40%
• Adverse Event 30%
• Pediatrics 30%
• Motor Neuron 30%
• Sitting 20%
• Spinal Muscular Atrophy 20%
• Infant 20%
• Arm 20%
• Survival 20%
• Follow up 10%
• Diseases 10%
• Outpatient 10%
• Infusion 10%
• Patient Population 10%
• Gene Therapy 10%
• Upper Respiratory Tract Infection 10%
• Growth 10%
• Autosomal Recessive Inheritance 10%
• Neuromuscular Disease 10%
• Drug 10%
• Exon 10%
• Intravenous Drug Administration 10%
• Point Mutation 10%
• Single Drug Dose 10%
• Respiratory Tract Infection 10%
• Alanine Aminotransferase 10%
• Gene 10%
• Chemotherapeutic Agent 10%
• Hypoxic Ischemic Encephalopathy 10%
• Intention-to-Treat Analysis 10%
• Fever 10%
• Assessment 10%
• Hospital 10%

Pharmacology, Toxicology and Pharmaceutical Science

• Onasemnogene Abeparvovec 70%
• Werdnig Hoffmann Disease 40%
• Adverse Event 30%
• Spinal Muscular Atrophy 20%
• Survival 20%
• Diseases 10%
• Respiratory Tract Infection 10%
• Drug 10%
• Fever 10%
• Alanine Aminotransferase 10%
• Hypoxic Ischemic Encephalopathy 10%
• Upper Respiratory Tract Infection 10%
• Neuromuscular Disease 10%
• Death 10%