A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease

Keyphrases

• Genetic Disease 100%
• Multi-domain 100%
• Mucopolysaccharidosis VII 100%
• Vestronidase Alfa 100%
• Responder Index 100%
• Ultrarare 100%
• Urinary GAGs 66%
• Drug Development 66%
• Treatment Effect 66%
• Ultra-rare Disease 66%
• Randomized Placebo-controlled Trial 33%
• Visual Acuity 33%
• Lysosomal Disease 33%
• Minimal Clinically Important Difference 33%
• Adverse Events 33%
• Statistical Power 33%
• Shoulder Flexion 33%
• Small Sample Size 33%
• Active Treatment 33%
• Heterogeneous Disease 33%
• Forced Vital Capacity 33%
• Pediatric Quality of Life Inventory 33%
• Dermatan Sulfate 33%
• Positive Treatment 33%
• Efficacy Analysis 33%
• Index Design 33%
• Sly Syndrome 33%
• Heterogeneous Studies 33%
• Bruininks-Oseretsky Test of Motor Proficiency 33%
• Multidimensional Fatigue Scale 33%
• Exposure-adjusted Incidence Rate 33%
• 6-minute Walk Test 33%
• Clinically Meaningful Improvement 33%
• Change from Baseline 33%

Medicine and Dentistry

• Genetic Disorder 100%
• Sly Syndrome 100%
• Vestronidase Alfa 100%
• Urinary System 66%
• Rare Disease 66%
• Treatment Effect 66%
• Diseases 66%
• Visual Acuity 33%
• Forced Vital Capacity 33%
• Crossing over 33%
• Pediatric Quality of Life Inventory 33%
• Dermatan Sulfate 33%
• Six Minute Walk Test 33%
• Placebo 33%
• Bruininks-Oseretsky Test of Motor Proficiency 33%
• Active Treatment 33%
• Adverse Event 33%