{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,23]],"date-time":"2026-09-23T02:25:13Z","timestamp":1790130313257,"version":"4.0.1"},"reference-count":7,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2019,12,2]],"date-time":"2019-12-02T00:00:00Z","timestamp":1575244800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2019,12,2]],"date-time":"2019-12-02T00:00:00Z","timestamp":1575244800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["EMBO Mol Med"],"published-print":{"date-parts":[[2020,1,9]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>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\u2010term 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\u2010associated tumors and discusses how these organizations have been collaborating and evolved practices to optimize funding and research support.<\/jats:p>","DOI":"10.15252\/emmm.201911656","type":"journal-article","created":{"date-parts":[[2019,12,2]],"date-time":"2019-12-02T21:10:54Z","timestamp":1575321054000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Funding community collaboration to develop effective therapies for neurofibromatosis type 1 tumors"],"prefix":"10.1038","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2010-4328","authenticated-orcid":false,"given":"Salvatore","family":"La Rosa","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vidya","family":"Browder","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annette C","family":"Bakker","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jaishri O","family":"Blakeley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sharad K","family":"Verma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6045-5344","authenticated-orcid":false,"given":"Ling M","family":"Wong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jill","family":"Morris","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Naba","family":"Bora","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2019,12,2]]},"reference":[{"key":"BFEMMM201911656_CR1","doi-asserted-by":"crossref","first-page":"818","DOI":"10.1002\/pbc.25896","volume":"63","author":"P de Blank","year":"2016","unstructured":"de Blank P, Kennedy M, Berman JI, Fisher MJ (2016) Systemic chemotherapy and white matter integrity in tracts associated with cognition among children with neurofibromatosis type 1. 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N Engl J Med 375: 2550\u20132560","journal-title":"N Engl J Med"},{"key":"BFEMMM201911656_CR4","doi-asserted-by":"crossref","first-page":"209","DOI":"10.1136\/jmedgenet-2018-105599","volume":"56","author":"IM Frayling","year":"2018","unstructured":"Frayling IM, Mautner V\u2010F, Van Minkelen R, Kallionpaa RA, Akta\u015f S, Baralle D, Ben\u2010Shachar\u00a0S, Callaway A, Cox H, Eccles DM et\u00a0al (2018) Breast cancer risk in neurofibromatosis\u00a0type 1 is a function of the type of, NF1, gene mutation: a new genotype\u2010phenotype correlation. J Med Genet 56: 209\u2013219","journal-title":"J Med Genet"},{"key":"BFEMMM201911656_CR5","doi-asserted-by":"crossref","first-page":"17004","DOI":"10.1038\/nrdp.2017.4","volume":"3","author":"DH Gutmann","year":"2017","unstructured":"Gutmann DH, Ferner RE, Listernick RH, Korf BR, Wolters PL, Johnson KJ (2017) Neurofibromatosis type 1. 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Washington, DC: NIH"},{"key":"BFEMMM201911656_CR9","unstructured":"National Institutes of Health (NIH) (2011) Estimates of funding for various research, condition, and disease categories (RCDC). 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Opinions, interpretations, conclusions, and recommendations are those of the author\/s and are not necessarily endorsed by the Department of Defense or by the National Institute of Health.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}}],"article-number":"EMMM201911656"}}