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
罕见病是公共卫生领域的优先事项。FDA has launched several programs and a final…
Jim Gallagher2024 年 2 月 21 日
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…
Danielle Pillsbury2023 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…
Danielle Pillsbury2023 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
全世界约有 400,000 名先天性肾上腺增生症（CAH）患者。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
