Expertise: Rare Disease 81 results
Rare Disease
Why Rare Disease Day Matters to Us
An estimated 300 million people globally suffer from a rare disease. Further still, 25 million people in the U.S. alone are living with an undiagnosed rare disease. Limitations in disease understanding and lack of approved treatments can mean countless challenges...
Developing a novel gene therapy requires a level of comfort with change and unpredictability. The path to regulatory approval is rarely straightforward, and roadblocks can enter at any turn. Premier Research was approached by a sponsor of an adeno-associated virus...
Rare Disease
Leslie Wetherell
Advancements in artificial intelligence, machine learning, and computing technologies have fueled progress in the use of sophisticated computational modeling and simulation as a tool for facilitating preclinical and clinical research in rare diseases. In this white paper, we explore the...
In a world of ever-increasing clinical trial costs, sponsors can feel overwhelming pressure to stretch their budgets without sacrificing the data they need for regulatory approval. In rare diseases, this pressure is intensified with the many challenges that often accompany...
Rare diseases are a distinctive and complex field within clinical development, characterized by significant hurdles from start to finish. Addressing these complexities requires innovative approaches to achieve a future of adaptability, quality, and efficiency. Here are 5 key steps to...