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…
CertaraJune 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…
CertaraJune 2, 2022
Leveraging Clinical Data from One Rare Disease to Support Drug Approval for Another
Atypical hemolytic uremic syndrome (aHUS) is an ultra-rare genetic disease that causes abnormal blood clot…
CertaraJune 1, 2022
A Bird’s Eye View on Rare Diseases: Key Drug Development Considerations for Thyroid Eye Disease
New therapeutics discovery and development for ocular diseases have been traditionally associated with a low…
CertaraDecember 15, 2021
Dose Optimization Using Population PK for an Orphan Drug
RAPALIMUS® Tablets (sirolimus) is Nobelpharma’s orphan drug developed from an oral medication that is sold…
CertaraNovember 17, 2021
