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
