Association of Lonafarnib Treatment vs No Treatment With Mortality Rate in Patients With Hutchinson-Gilford Progeria Syndrome

IMPORTANCE: Hutchinson-Gilford progeria syndrome (HGPS) is an extremely rare fatal premature aging disease. There is no approved treatment. OBJECTIVE: To evaluate the association of monotherapy using the protein farnesyltransferase inhibitor lonafarnib with mortality rate in children with HGPS. DESI...

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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:JAMA
Prif Awduron: Gordon, Leslie B., Shappell, Heather, Massaro, Joe, D’Agostino, Ralph B., Brazier, Joan, Campbell, Susan E., Kleinman, Monica E., Kieran, Mark W.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Medical Association 2018
Pynciau:
Preliminary Communication
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC5933395/
https://ncbi.nlm.nih.gov/pubmed/29710166
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/jama.2018.3264
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