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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1953 1
1966 3
1967 1
1969 1
1970 3
1971 3
1972 3
1973 3
1974 3
1975 5
1976 2
1977 2
1978 2
1979 3
1980 2
1981 6
1982 3
1983 1
1984 5
1985 5
1986 4
1987 4
1989 3
1990 4
1991 3
1992 3
1993 6
1995 3
1996 6
1997 8
1998 1
1999 11
2000 8
2001 9
2002 13
2003 11
2004 10
2005 13
2006 15
2007 18
2008 17
2009 19
2010 28
2011 33
2012 32
2013 39
2014 31
2015 31
2016 34
2017 31
2018 30
2019 23
2020 53
2021 50
2022 31
2023 25
2024 15

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653 results

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Quoted phrase not found in phrase index: "Cafe-au-lait macules with pulmonary stenosis"
Page 1
Current Understanding of Neurofibromatosis Type 1, 2, and Schwannomatosis.
Tamura R. Tamura R. Int J Mol Sci. 2021 May 29;22(11):5850. doi: 10.3390/ijms22115850. Int J Mol Sci. 2021. PMID: 34072574 Free PMC article. Review.
This study discusses molecular pathways and related targeted therapies for NF1, NF2, and SWN and reviews recent clinical trials which involve NF patients....
This study discusses molecular pathways and related targeted therapies for NF1, NF2, and SWN and reviews recent clinical trials which …
Selumetinib in Children with Inoperable Plexiform Neurofibromas.
Gross AM, Wolters PL, Dombi E, Baldwin A, Whitcomb P, Fisher MJ, Weiss B, Kim A, Bornhorst M, Shah AC, Martin S, Roderick MC, Pichard DC, Carbonell A, Paul SM, Therrien J, Kapustina O, Heisey K, Clapp DW, Zhang C, Peer CJ, Figg WD, Smith M, Glod J, Blakeley JO, Steinberg SM, Venzon DJ, Doyle LA, Widemann BC. Gross AM, et al. N Engl J Med. 2020 Apr 9;382(15):1430-1442. doi: 10.1056/NEJMoa1912735. Epub 2020 Mar 18. N Engl J Med. 2020. PMID: 32187457 Free PMC article. Clinical Trial.
BACKGROUND: No approved therapies exist for inoperable plexiform neurofibromas in patients with neurofibromatosis type 1. METHODS: We conducted an open-label, phase 2 trial of selumetinib to determine the objective response rate among patients with plexiform neurofibromas …
BACKGROUND: No approved therapies exist for inoperable plexiform neurofibromas in patients with neurofibromatosis type 1. METHODS: We conduc …
Neurofibromatosis type 1: a multidisciplinary approach to care.
Hirbe AC, Gutmann DH. Hirbe AC, et al. Lancet Neurol. 2014 Aug;13(8):834-43. doi: 10.1016/S1474-4422(14)70063-8. Lancet Neurol. 2014. PMID: 25030515 Free article. Review.
Although many manifestations of neurofibromatosis type 1 affect the nervous system, other organs and tissues can also be affected. Because of the varying features and clinical heterogeneity inherent to this disorder, patients can present to different medical and surgical s …
Although many manifestations of neurofibromatosis type 1 affect the nervous system, other organs and tissues can also be affected. Because o …
NF1, Neurofibromin and Gene Therapy: Prospects of Next-Generation Therapy.
Cui XW, Ren JY, Gu YH, Li QF, Wang ZC. Cui XW, et al. Curr Gene Ther. 2020;20(2):100-108. doi: 10.2174/1566523220666200806111451. Curr Gene Ther. 2020. PMID: 32767931 Review.
Neurofibromatosis type 1 [NF1] is an autosomal dominant genetic disorder affecting multiple organs. NF1 is well known for its various clinical manifestations, including cafe-au-late macules, Lisch nodules, bone deformity and neurofibromas. However, the …
Neurofibromatosis type 1 [NF1] is an autosomal dominant genetic disorder affecting multiple organs. NF1 is well known for its various cli
Treatment decisions and the use of MEK inhibitors for children with neurofibromatosis type 1-related plexiform neurofibromas.
Armstrong AE, Belzberg AJ, Crawford JR, Hirbe AC, Wang ZJ. Armstrong AE, et al. BMC Cancer. 2023 Jun 16;23(1):553. doi: 10.1186/s12885-023-10996-y. BMC Cancer. 2023. PMID: 37328781 Free PMC article. Review.
Selumetinib is currently the only licensed medical therapy indicated for use in pediatric patients with symptomatic, inoperable NF1-PN, with approval based on the results of this pivotal clinical study. Several other MEK inhibitors (binimetinib, mirdametinib, trametinib) a …
Selumetinib is currently the only licensed medical therapy indicated for use in pediatric patients with symptomatic, inoperable NF1-PN, with …
A Review of Selumetinib in the Treatment of Neurofibromatosis Type 1-Related Plexiform Neurofibromas.
Anderson MK, Johnson M, Thornburg L, Halford Z. Anderson MK, et al. Ann Pharmacother. 2022 Jun;56(6):716-726. doi: 10.1177/10600280211046298. Epub 2021 Sep 18. Ann Pharmacother. 2022. PMID: 34541874 Review.
DATA SYNTHESIS: The open-label, multicenter, single-arm, phase II SPRINT trial demonstrated clinically significant improvements in PN-related complications. ...CONCLUSION: Selumetinib exhibits impressive antitumor activity and sustained clinical benefit in patients …
DATA SYNTHESIS: The open-label, multicenter, single-arm, phase II SPRINT trial demonstrated clinically significant improvements in PN …
MEK Inhibition Synergizes with TYK2 Inhibitors in NF1-Associated Malignant Peripheral Nerve Sheath Tumors.
Borcherding DC, Amin NV, He K, Zhang X, Lyu Y, Dehner C, Bhatia H, Gothra A, Daud L, Ruminski P, Pratilas CA, Pollard K, Sundby T, Widemann BC, Hirbe AC. Borcherding DC, et al. Clin Cancer Res. 2023 Apr 14;29(8):1592-1604. doi: 10.1158/1078-0432.CCR-22-3722. Clin Cancer Res. 2023. PMID: 36799629 Free PMC article.
CONCLUSIONS: These data provide the preclinical rationale for the development of a phase I clinical trial of deucravacitinib and mirdametinib in NF1-assosciated MPNST....
CONCLUSIONS: These data provide the preclinical rationale for the development of a phase I clinical trial of deucravacitinib a …
Long-term safety and efficacy of selumetinib in children with neurofibromatosis type 1 on a phase 1/2 trial for inoperable plexiform neurofibromas.
Gross AM, Dombi E, Wolters PL, Baldwin A, Dufek A, Herrera K, Martin S, Derdak J, Heisey KS, Whitcomb PM, Steinberg SM, Venzon DJ, Fisher MJ, Kim A, Bornhorst M, Weiss BD, Blakeley JO, Smith MA, Widemann BC. Gross AM, et al. Neuro Oncol. 2023 Oct 3;25(10):1883-1894. doi: 10.1093/neuonc/noad086. Neuro Oncol. 2023. PMID: 37115514 Free PMC article. Clinical Trial.
BACKGROUND: Selumetinib shrank inoperable symptomatic plexiform neurofibromas (PN) in children with neurofibromatosis type 1 (NF1) and provided clinical benefit for many in our previously published phase 1/2 clinical trials (SPRINT, NCT01362803). ...
BACKGROUND: Selumetinib shrank inoperable symptomatic plexiform neurofibromas (PN) in children with neurofibromatosis type 1 (NF1) and provi …
Novel molecular targeted therapies for patients with neurofibromatosis type 1 with inoperable plexiform neurofibromas: a comprehensive review.
Solares I, Viñal D, Morales-Conejo M, Rodriguez-Salas N, Feliu J. Solares I, et al. ESMO Open. 2021 Aug;6(4):100223. doi: 10.1016/j.esmoop.2021.100223. Epub 2021 Aug 10. ESMO Open. 2021. PMID: 34388689 Free PMC article. Review.
Systemic therapies had not succeeded to reduce PN tumor volume until 2016 when the first trial with an MAPK/extracellular-signal-regulated kinase (MEK) inhibitor was published. ...
Systemic therapies had not succeeded to reduce PN tumor volume until 2016 when the first trial with an MAPK/extracellular-signal-regu …
653 results