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 Pillsbury2024年12月4日
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 Pillsbury2024年7月11日
How to Navigate the FDA Landscape for an Orphan Drug
希少疾患は公衆衛生上の優先事項です。FDA has launched several programs and a final…
Certara2024年2月21日
Methods to Increase Signal Finding in Rare Disease Drug Development
希少疾患の治療薬に関する規制状況は大きく変化しています。Gleaning from several…
Certara2023年9月8日
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…
Certara2023年2月3日
Exceling at Accelerating in Rare Disease Drug Development
By: Rajesh Krishna, PhD, FAAPS and Steve Sibley According to the National Institutes of Health,…
Certara2023年1月26日
Simcyp Simulator Helps Optimize Drug Dosing in Adults & Adolescents with Orphan Disease
先天性副腎過形成症（CAH）は、世界中でおよそ400,000人の患者さんがいます。Current therapy for CAH uses a…
Becca Bucci2022年6月6日
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 Bucci2022年6月2日
