Open AccessAssociation of Lonafarnib Treatment vs No Treatment With Mortality Rate in Patients With Hutchinson-Gilford Progeria Syndrome
Author(s) -
Leslie B. Gordon,
Heather Shappell,
Joseph M. Massaro,
Ralph B. D’Agostino,
Joan F. Brazier,
Susan Campbell,
Monica E. Kleinman,
Mark W. Kieran
Publication year - 2018
Publication title -
jama
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 4.688
H-Index - 680
eISSN - 1538-3598
pISSN - 0098-7484
DOI - 10.1001/jama.2018.3264
Subject(s) - medicine , premature aging , lmna , progeria , cohort , pediatrics , physiology , lamin , biochemistry , chemistry , nucleus , psychiatry , gene
Hutchinson-Gilford progeria syndrome (HGPS) is an extremely rare fatal premature aging disease. There is no approved treatment.
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