Publications

Evaluating the clinical benefit of interventions aimed at treating conditions with heterogeneous symptom....

Panel discussion at the National Organization of Rare Disorders (NORD) Breakthrough Summit in Washington, DC, held October 15 –

The Critical Path Institute’s Polycystic Disease Outcomes Consortium, including key stakeholders (patients, medical experts

Autosomal recessive polycystic kidney disease (ARPKD) is a rare hepatorenal fibrocystic disease that often presents prenatally

Inherited cystic diseases include a spectrum of disorders, such as autosomal dominant polycystic kidney disease (ADPKD), autosomal

Autosomal dominant polycystic kidney disease (ADPKD) is a major genetic disorder affecting up to 12 million individuals worldwide