Rare and Orphan Diseases

Critical Path for Alpha-1 Antitrypsin Deficiency

Rare and Orphan Diseases Pediatrics

Critical Path for Lysosomal Diseases Pre-Consortium

Rare and Orphan Diseases Pediatrics

Critical Path for Rare Neurodegenerative Diseases

Neuroscience Rare and Orphan Diseases

Critical Path to Therapeutics for the Ataxias

Neuroscience Rare and Orphan Diseases

Duchenne Regulatory Science Consortium

Neuroscience Rare and Orphan Diseases Pediatrics

Polycystic Kidney Disease Outcomes Consortium

Rare and Orphan Diseases Pediatrics

Rare Disease Cures Accelerator-Data and Analytics Platform

Rare and Orphan Diseases

Rare Disease Clinical Outcome Assessment Consortium

Core Competencies Rare and Orphan Diseases Pediatrics Clinical Outcome Assessment

Huntington’s Disease Regulatory Science Consortium

Neuroscience Rare and Orphan Diseases