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…
Danielle Pillsbury2020 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…
Danielle Pillsbury2019 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…
Danielle Pillsbury2017 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…
Danielle Pillsbury2016 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…
Danielle Pillsbury2016 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…
Danielle Pillsbury2015 年 2 月 15 日
