Gorlin Syndrome Treatment Burden Problem: What the Patidegib Readout Could Change
Patients with severe Gorlin syndrome can face repeated surgeries year after year, many of them on the face. Each one can leave a scar, and a face only has so much tissue to spare.Most people meet basa ...
The Evolution of HEOR in Rare Diseases: From Cost-Effectiveness to Strategic Market Access
There used to be a simple rule in pharma: scientists built the drug, clinical teams proved it worked, and health economists showed up near the end to do the math. Run the trial, demonstrate efficacy, ...