The Promises and Pitfalls of Gene Therapy for Rare Diseases
The state of gene therapy faces much uncertainty as recent regulatory and clinical setbacks have…
Jim Gallagher2021年4月29日
The Rare Disease Drug Dilemma: Great Value, High Price, Limited Access?
This webinar will explain how pricing has evolved for the rare disease drug market and…
webdev2020年2月19日
Innovative Solutions for Fast-Tracking Drugs for Rare Diseases
This webinar discussed which pharmaceutical payer strategies work and which don't in our rapidly changing…
webdev2019年8月10日
Obeticholic Acid—From PK Model to Drug Label
Watch this webinar with Jeffrey Edwards to learn how he used physiologic pharmacokinetic modeling to…
webdev2017年6月1日
Rare Disease and Orphan Drug Development
There are 7,000 rare diseases impacting 350 million patients worldwide, yet only 300 of these…
webdev2016年8月3日
Population Pharmacokinetic and Pharmacodynamic Analyses from a 4-Month Intra-dose Escalation and Its Subsequent 12-month Dose Titration Studies for a Human Monoclonal Anti-FGF23 Antibody (KRN23) in Adults with X-linked Hypophosphatemia
X-linked hypophosphatemia (XLH) is an inherited metabolic bone disease with abnormally elevated serum FGF23 resulting…
webdev2016年4月1日
Chemotherapy of Second Stage Human African Trypanosomiasis: Comparison Between the Parenteral Diamidine DB829 and Its Oral Prodrug DB868 in Vervet Monkeys
Human African trypanosomiasis (HAT, sleeping sickness) ranks among the most neglected tropical diseases based on…
webdev2015年2月15日
