Quantitative Systems Pharmacology-Based Digital Twins Approach Supplements Clinical Trial Data for Enzyme Replacement Therapies in Pompe Disease
Pompe disease is a rare, progressive neuromuscular disease caused by deficient lysosomal glycogen degradation, and…
Danielle PillsburyDecember 4, 2024
New Horizons of Model Informed Drug Development in Rare Diseases Drug Development
Model-informed drug development is revolutionizing the way we approach rare diseases, where traditional large-scale clinical…
Danielle PillsburyJuly 11, 2024
How to Navigate the FDA Landscape for an Orphan Drug
Rare diseases are a public health priority. FDA has launched several programs and a final…
CertaraFebruary 21, 2024
Methods to Increase Signal Finding in Rare Disease Drug Development
The regulatory landscape for drugs to treat rare diseases is being disrupted. Gleaning from several…
CertaraSeptember 8, 2023
Artificial Intelligence: What is it & how can it accelerate rare disease drug development?
Large & complex information streams present opportunities for the AI data scientist to aid in…
CertaraFebruary 3, 2023
Exceling at Accelerating in Rare Disease Drug Development
By: Rajesh Krishna, PhD, FAAPS and Steve Sibley According to the National Institutes of Health,…
CertaraJanuary 26, 2023
Simcyp Simulator Helps Optimize Drug Dosing in Adults & Adolescents with Orphan Disease
Congenital adrenal hyperplasia (CAH) affects about 400,000 patients worldwide. Current therapy for CAH uses a…
Becca BucciJune 6, 2022
PBPK Modeling in Adults Enables DDI Risk Assessment in Children with SMA
In 2020, the US FDA approved Risdiplam (Evrysdi®) as the first orally administered drug to…
Becca BucciJune 2, 2022
