Sanfilippo Syndrome Type A
Pipeline by Development Stage
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Clinical Trials (8)
Total enrollment: 689 patients across 8 trials
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Taste Assessment Study of SHP429 in Healthy Adult Subjects
Value of PET/MR Enterography in the Assessment of Crohn's Disease Using a Collagen-binding Radiotracer.
Assessment of Updated Educational Materials for Instanyl®
Assessment of Immune Activation and Tolerance in Celiac Disease During Gluten Challenge
Sophisticated Assessment of Disease Burden in Patients With Fabry Disease
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®