Blog
Decoding FDA Gene Therapy Guidances Part Three: Long-term Follow-ups
In Part Three of our Decoding FDA Gene Therapy Guidances series, we discuss the need for thorough long-term follow-up studies for these products, as…
All Results
Blog
Blog
Decoding FDA Gene Therapy Guidances Part Two: Rare Disease
In Part Two of Decoding FDA Gene Therapy Guidances, we discuss three separate guidances that provide further insight into how sponsors can safely and…
Blog
Decoding FDA Gene Therapy Guidances Part One: Chemistry, Manufacturing, and Control
In Part One of our Decoding FDA Gene Therapy Guidances series, we discuss the significance of several new FDA guidances regarding human gene therapy…
Blog
Ask the Experts: What Does the Future Hold for CRISPR Gene Editing Technology?
Changing a living organism’s DNA sounds like something out of science fiction, but recent strides in genome (gene) editing have given scientists…
Blog
Ask the Experts: Why Do Rare Disease Clinical Trials Require a Unique Approach?
According to the National Organization for Rare Disorders (NORD), 90% of rare disorders do not have an FDA-approved treatment. We spoke with Juliane…
Blog
HEALEY ALS Platform Trial Drives Innovation in ALS Clinical Research
Amyotrophic lateral sclerosis (ALS) is a lethal, relentlessly progressive neurodegenerative disease, and the most common adult-onset motor neuron…
White Paper
Partnering with Patients to Develop Meaningful Endpoints – Practical Considerations
Selection of appropriate endpoints for rare disease clinical studies can be challenging but is critical to assessing the efficacy of new medicines.…
White Paper
Decentralized Trials: Reshaping the Rare Disease Landscape
Clinical drug development is moving away from traditional approaches that rely on rare disease patients to travel long distances to clinical sites…
Blog
CRISPR Technology Offers New Hope for Patients with Devastating Diseases
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is a powerful new gene-editing tool with unprecedented implications for the future…
Newsletter
Rare Diseases Newsletter
This quarterly publication strives to keep you up to date on PRA’s Center for Rare Diseases initiatives and activities. Interested in learning more…