WODC Panel Summary: Pricing & Reimbursement for Rare Diseases in the United States
WODC Panel Summary: Outlook on European Market Access Strategies for Rare Diseases
The Future of Reimbursement for Cell & Gene Therapies
Precision ADVANCE at ISPOR 2021
Building the first-of-its kind gene therapy facility, commercializing one of the first gene therapies and industrializing the largest gene therapy footprint in the world
Navigating the CAR T-cell Therapy Landscape to Develop Market Access and Distribution Strategies
Process and Communication: Navigating Logistical Complexity in Matched Cellular Therapy Trials
Mining Stakeholder Insights on Access to Gene Therapies for Hemophilia
The Value of Gene Therapy in Spinal Muscular Atrophy
Ensuring Provider Education And Readiness For Using A Novel CAR-T
What’s Next for Cell and Gene Therapies in 2023 and Beyond
State of the Union for Advanced Medicines
Considerations for the Clinical Development of Cell & Gene Therapies (Part 1: Cell Therapies)
Considerations for the Clinical Development of Cell & Gene Therapies (Part 2: Gene Therapies)
Innovation: Shortening the Path to Commercialization in Advanced Therapies
The Great Debate: Internal vs. External Manufacturing for Advanced Therapies
The Future of Reimbursement for Cell and Gene Therapies
Gene Therapies in Hemophilia
Considerations for Early-Stage Gene Therapy Start-ups: From Clinical Development to Manufacturing to Commercialization
Delivering on the Promise of Cell Therapy: Challenges and Trends
Optimizing Successful Development of Viral Vector Gene Therapies, Gene Therapy Trials, and Companion Diagnostics
Perspectives on Gene Therapy: Defining and Demonstrating Value to Payers
Advantages of Using POD Cleanroom Technology
What to Consider When Conducting a “Make vs. Buy” Analysis for a Cell & Gene Therapy Facility
Developing Advanced Therapies: Considerations for Internal Versus External Manufacturing
Costs and health resource use in patients with X-linked myotubular myopathy: insights from U.S. commercial claims. Journal of Managed Care & Specialty Pharmacy. 2021.
An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER). Journal of Market Access & Health Policy. 2020.
Economic Burden of X-Linked Myotubular Myopathy (XLMTM) By Ventilation Status. ISPOR 25th Annual International Meeting. Orlando FL. 2020.
Healthcare resource use and expenditures in patients with X-linked myotubular myopathy (XLMTM). ISPOR 25th Annual International Meeting. Orlando FL. 2020.
Health resource use in patients with x-linked myotubular myopathy (XLMTM): Data from the RECENSUS Study. ISPOR 25th Annual International Meeting. Orlando FL. 2020.
Recommendations for the Development of Cell-Based Anti-Viral Vector Neutralizing Antibody Assays. AAPS Journal. 2020.
Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients. Journal of Market Access & Health Policy. 2019.