Rare Disease
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Bringing the Power of Genetics to a Rare Disease Gene Therapy Program
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1:03:56
Webinar: Gene Therapy for Rare and Ultra-rare Diseases
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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.
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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.
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56:41
Rare & Orphan Disease Endpoints: Challenges & Examples from Neurodevelopment
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