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Sirolimus Improves Pain in NF1 Patients With Severe Plexiform Neurofibromas

✍ Scribed by Hua, C.; Zehou, O.; Ducassou, S.; Minard-Colin, V.; Hamel-Teillac, D.; Wolkenstein, P.; Valeyrie-Allanore, L.

Book ID
125496915
Publisher
American Academy of Pediatrics
Year
2014
Tongue
English
Weight
953 KB
Volume
133
Category
Article
ISSN
0031-4005

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Neurofibromatosis 1 (NF1) is an autosomal dominant disorder caused by genetic alterations of the NF1 gene on 17q11.2. About 30% of NF1 patients develop plexiform neurofibromas (PNFs), which often cause severe clinical deficits. To determine whether there is a certain genotype underlying PNFs or subt