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

Keyphrases

• Gene Therapy 100%
• Phase II Trial 100%
• Infantile Onset 100%
• Onasemnogene Abeparvovec 100%
• Spinal muscular Atrophy Type 1 100%
• Adverse Events 42%
• Pediatric 42%
• Clinical Research 42%
• Spinal muscular Atrophy 28%
• Natural History 28%
• Biallelic 28%
• Aging Studies 28%
• Survival Motor Neuron 28%
• Patient-completed 14%
• Pyrexia 14%
• Autosomal Recessive 14%
• Point mutation 14%
• Patient Demographics 14%
• Severe Disease 14%
• Intravenous Infusion 14%
• Alanine Aminotransferase 14%
• Motor Neuron Disease 14%
• Exon 14%
• Long-term Safety 14%
• Respiratory Infection 14%
• Hypoxic-ischemic Brain Injury 14%
• Upper Respiratory Tract Infection 14%
• Intention-to-treat 14%
• Neuromuscular Disease 14%
• Outpatient Follow-up 14%
• Novartis 14%
• France 14%
• Belgium 14%
• Italy 14%
• Untreated Patients 14%
• Ventilatory Support 14%
• Deletion mutation 14%
• Research Cohort 14%
• Safety Signal 14%
• Early Termination 14%
• Vector Genomes 14%
• SMN2 14%
• Gene Replacement Therapy 14%
• EU Studies 14%
• Growth References 14%

Medicine and Dentistry

• Arm 100%
• Gene Therapy 100%
• Onasemnogene Abeparvovec 100%
• Werdnig Hoffmann Disease 100%
• Adverse Event 42%
• Pediatrics 42%
• Clinical Research 42%
• Spinal Muscular Atrophy 28%
• Sitting 28%
• Infusion 14%
• Fever 14%
• Exon 14%
• Point Mutation 14%
• Intention-to-Treat Analysis 14%
• Intravenous Drug Administration 14%
• Outpatient 14%
• Patient Population 14%
• Respiratory Tract Infection 14%
• Autosomal Recessive Inheritance 14%
• Single Drug Dose 14%
• Alanine Aminotransferase 14%
• Upper Respiratory Tract Infection 14%
• Neuromuscular Disease 14%
• Hypoxic Ischemic Encephalopathy 14%
• Gene Replacement Therapy 14%
• Diseases 14%

Pharmacology, Toxicology and Pharmaceutical Science

• Werdnig Hoffmann Disease 100%
• Onasemnogene Abeparvovec 100%
• Adverse Event 42%
• Clinical Research 42%
• Spinal Muscular Atrophy 28%
• Fever 14%
• Replacement Therapy 14%
• Respiratory Tract Infection 14%
• Alanine Aminotransferase 14%
• Upper Respiratory Tract Infection 14%
• Neuromuscular Disease 14%
• Hypoxic Ischemic Encephalopathy 14%
• Diseases 14%