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Systemic Therapy Development in Von Hippel-Lindau Disease: An Outsized Contribution from an Orphan Disease.


ABSTRACT: Over the last several decades, an improved understanding of von Hippel-Lindau disease and its underlying biology has informed the successful development of numerous anti-cancer agents, particularly for the treatment of advanced renal cell carcinoma. Most recently, this has culminated in the first regulatory approval for a systemic therapy for VHL disease-associated neoplasms. This review will trace the clinical development of systemic therapies for VHL disease and additionally highlight anticipated challenges and opportunities for future VHL systemic therapy.

SUBMITTER: Narayan V 

PROVIDER: S-EPMC9658616 | biostudies-literature | 2022 Oct

REPOSITORIES: biostudies-literature

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Systemic Therapy Development in Von Hippel-Lindau Disease: An Outsized Contribution from an Orphan Disease.

Narayan Vivek V   Jonasch Eric E  

Cancers 20221028 21


Over the last several decades, an improved understanding of von Hippel-Lindau disease and its underlying biology has informed the successful development of numerous anti-cancer agents, particularly for the treatment of advanced renal cell carcinoma. Most recently, this has culminated in the first regulatory approval for a systemic therapy for VHL disease-associated neoplasms. This review will trace the clinical development of systemic therapies for VHL disease and additionally highlight anticipa  ...[more]

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