Rare Disease
Expertise: Rare Disease 84 results
Progress in advanced computational methods using modeling and simulation has been enabled by advancements in computing technologies and the rise of artificial intelligence and machine learning. This has opened new opportunities in pharmaceutical drug development, such as the ability to...
There is increasing focus on the development of new cell and gene therapies (CGT) in rare disease.
In the complex and uncharted territory of rare disease research, particularly cell and gene studies, Premier offers significant experience with natural history studies, precision medicine, and real-world data planning, trial design, and regulatory strategy to guide rare disease product development.
Small patient populations. Limited data on disease. Patient enrollment and retention challenges. Narrow site pools. These are some of the most common hurdles sponsors face in rare disease drug development. The right CRO partner can help turn these challenges into...
Small patient populations. Geographic dispersion. Competing studies. Sound familiar? If you’re a sponsor of a rare disease study, you know all too well the challenges that come with recruiting patients. These challenges lead to immense pressure to design and implement...
As industry interest in rare diseases increases, so does competition within an already challenging clinical research environment where patient pools are inherently limited and often geographically dispersed. To differentiate their trials and inspire interest among patients and other key stakeholders,...
MORRISVILLE, N.C., MILAN, ITALY, TRIESTE, ITALY, March 13, 2023 — Premier Research, whose mission is to help the most innovative biotech and device companies take their best ideas from concept to commercialization, and InSilicoTrials, a leader in the use of artificial...
Individually, rare diseases by definition have a low prevalence, but collectively, the societal burden and impact of these uncommon conditions is significant. The majority of rare diseases are genetic in origin, and advances in genomic sequencing tools and technologies have...