Funding community collaboration to develop effective therapies for neurofibromatosis type 1 tumors

Salvatore La Rosa, Vidya Browder, Annette C. Bakker, Jaishri O. Blakeley, Sharad K. Verma, Ling M. Wong, Jill Morris, Naba Bora

Research output: Contribution to journalComment/debate

Abstract

The time from identifying a drug target to a new drug approval is often measured in decades and can take even longer for therapies to treat rare diseases. In fact, 95% of rare diseases do not have a specific therapy approved at all. Coordinated efforts to augment the drug development pipeline along with long-term and comprehensive support that enable scientific breakthroughs for rare diseases are possible, but it requires integration across multiple stakeholders. This article analyzes the coordinated funding efforts of four federal and philanthropic organizations to advance drug development for neurofibromatosis type 1-associated tumors and discusses how these organizations have been collaborating and evolved practices to optimize funding and research support.

Original languageEnglish (US)
Article numbere11656
JournalEMBO Molecular Medicine
DOIs
StateAccepted/In press - Jan 1 2019

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Neurofibromatosis 1
Rare Diseases
Organizations
Pharmaceutical Preparations
Drug Approval
Neoplasms
Therapeutics

ASJC Scopus subject areas

  • Molecular Medicine

Cite this

Funding community collaboration to develop effective therapies for neurofibromatosis type 1 tumors. / La Rosa, Salvatore; Browder, Vidya; Bakker, Annette C.; Blakeley, Jaishri O.; Verma, Sharad K.; Wong, Ling M.; Morris, Jill; Bora, Naba.

In: EMBO Molecular Medicine, 01.01.2019.

Research output: Contribution to journalComment/debate

La Rosa, Salvatore ; Browder, Vidya ; Bakker, Annette C. ; Blakeley, Jaishri O. ; Verma, Sharad K. ; Wong, Ling M. ; Morris, Jill ; Bora, Naba. / Funding community collaboration to develop effective therapies for neurofibromatosis type 1 tumors. In: EMBO Molecular Medicine. 2019.
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