
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…
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…
In Part Two of Decoding FDA Gene Therapy Guidances, we discuss three separate guidances that provide further insight into how sponsors can safely and…
In Part One of our Decoding FDA Gene Therapy Guidances series, we discuss the significance of several new FDA guidances regarding human gene therapy…
Changing a living organism’s DNA sounds like something out of science fiction, but recent strides in genome (gene) editing have given scientists…
According to the National Organization for Rare Disorders (NORD), 90% of rare disorders do not have an FDA-approved treatment. We spoke with Juliane…
Amyotrophic lateral sclerosis (ALS) is a lethal, relentlessly progressive neurodegenerative disease, and the most common adult-onset motor neuron…
Selection of appropriate endpoints for rare disease clinical studies can be challenging but is critical to assessing the efficacy of new medicines.…
Clinical drug development is moving away from traditional approaches that rely on rare disease patients to travel long distances to clinical sites…
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is a powerful new gene-editing tool with unprecedented implications for the future…
This quarterly publication strives to keep you up to date on PRA’s Center for Rare Diseases initiatives and activities. Interested in learning more…