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New Rare Disease Drugs and Research Advancements

XTalks

FEBRUARY 27, 2024

Rare Disease Day 2024, which falls on February 29 this year, is an opportunity to unite under a common cause: to bring attention to the challenges faced by those living with rare diseases and to push for advancements in research, treatment and policy. Sarepta hopes to clarify Elvidys’ effectiveness in older children in a confirmatory trial.

Research

Research Drugs Gene Therapy Clinical Trials

Amtagvi (Lifileucel) Scores Landmark Win as First FDA-Approved T-Cell Therapy for a Solid Tumor

XTalks

FEBRUARY 27, 2024

Addressing the high price tag, Iovance’s commercial chief Jim Ziegler said, “Payers have expressed their appreciation for the value proposition for Amtagvi.” Amtagvi Considerations Amtagvi’s approval was decades in the making, with TIL therapy dating back to research conducted at the National Institutes of Health (NIH) in the 1980s.

FDA Approval

FDA Approval Manufacturing In-Vivo Trials

New Rare Disease Drugs and Research Advancements

Amtagvi (Lifileucel) Scores Landmark Win as First FDA-Approved T-Cell Therapy for a Solid Tumor

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