Rare Disease
-
Read Flipbook
Bringing the Power of Genetics to a Rare Disease Gene Therapy Program
-
1:03:56Watch VideoWebinar: Gene Therapy for Rare and Ultra-rare Diseases
-
Read Flipbook
Rare Disease Case Study: Pediatric Sickle Cell Anemia
Our client, a Top 10 pharmaceutical company, needed to boost enrollment for their phase ll pediatric sickle cell anemia study.
-
Read Flipbook
Rare Disease Case Study: Familial Amyloidosis
Our client, a top-tier CRO, was faced with the challenge of enrolling for a non-interventional screening study in the familial amyloidotic cardiomyopathy (FAC) patient population.
-
56:41Watch VideoRare & Orphan Disease Endpoints: Challenges & Examples from Neurodevelopment
-
Loading More...
