Advancing Therapies for Neurofibromatosis Type 1 (NF1): Lessons Learned from Every Patient

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07/02/23

Air date: Wednesday, December 8, 2021, 3PM
Runtime: 01: 01 :09
Description: WALS Annual Astute Clinician Lecture


The Astute Clinician Lecture, established in 1998 through a gift from the late Dr. Robert W. Miller and his wife, Haruko, recognizes U.S. scientists who have observed unusual clinical occurrences and, by investigating them, have opened an important new avenue of research.

Dr. Widemann is Deputy Clinical Director for the NCI Center for Cancer Research, chief of its Pediatric Oncology Branch, and head of its Pharmacology and Experimental Therapeutics Section. Trained as a pediatric oncologist with expertise in drug development, she has applied her expertise to study genetic tumor predisposition syndromes, in particular neurofibromatosis type 1 (NF1) and very rare pediatric and adult solid tumors.

Widemann's pioneering research on NF1 resulted in the first U.S. Food and Drug Administration approved medical therapy, the MEK inhibitor selumetinib, for children with NF1 and inoperable, symptomatic plexiform neurofibromas in 2020. For the Astute Clinician Lecture, she will describe her lab's recent successes as well as her current and future direction.

Refer to https://irp.nih.gov/pi/brigitte-widemann for an overview of her research.

Co-sponsored by the NIH Clinical Center.

For more information go to https://oir.nih.gov/wals

Author: Brigitte C. Widemann, M.D., Deputy Clinical Director, Center for Cancer Research, Chief of Pediatric Oncology Branch, Head of Pharmacology and Experimental Therapeutics Section, NCI, NIH

Permanent link: https://videocast.nih.gov/watch=43814

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