Rare and Orphan Diseases

C-Path’s rare and orphan disease program unites regulators, pharmaceutical and biotech companies, academics, and patient advocates to advance drug development in diseases such as Duchenne muscular dystrophy, Huntington’s disease, polycystic kidney disease, and rare neurodegenerative 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

Huntington’s Disease Regulatory Science Consortium

Neuroscience Rare and Orphan Diseases

Polycystic Kidney Disease Outcomes Consortium

Rare and Orphan Diseases Pediatrics

Rare Disease Clinical Outcome Assessment Consortium

Core Competencies Rare and Orphan Diseases Pediatrics Clinical Outcome Assessment

Rare Disease Cures Accelerator-Data and Analytics Platform

Rare and Orphan Diseases