Progeria medical therapy

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Vamsikrishna Gunnam M.B.B.S [2]


There is no treatment for Hutchinson-Gilford progeria syndrome (HGPS); the mainstay of therapy is supportive care. But the good news is that there are new investigational therapies for Hutchinson-Gilford progeria syndrome (HGPS) patients which include lonafarnib and everolimus.

Medical Therapy




  1. Adam MP, Ardinger HH, Pagon RA, Wallace SE, Bean LJH, Stephens K; et al. (1993). "GeneReviews®". PMID 20301300.
  2. Adam MP, Ardinger HH, Pagon RA, Wallace SE, Bean LJH, Stephens K; et al. (1993). "GeneReviews®". PMID 20301300.
  3. Gordon LB, Kleinman ME, Miller DT, Neuberg DS, Giobbie-Hurder A, Gerhard-Herman M; et al. (2012). "Clinical trial of a farnesyltransferase inhibitor in children with Hutchinson-Gilford progeria syndrome". Proc Natl Acad Sci U S A. 109 (41): 16666–71. doi:10.1073/pnas.1202529109. PMC 3478615. PMID 23012407.
  4. Ullrich NJ, Kieran MW, Miller DT, Gordon LB, Cho YJ, Silvera VM; et al. (2013). "Neurologic features of Hutchinson-Gilford progeria syndrome after lonafarnib treatment". Neurology. 81 (5): 427–30. doi:10.1212/WNL.0b013e31829d85c0. PMC 3776537. PMID 23897869.
  5. Gordon LB, Massaro J, D'Agostino RB, Campbell SE, Brazier J, Brown WT; et al. (2014). "Impact of farnesylation inhibitors on survival in Hutchinson-Gilford progeria syndrome". Circulation. 130 (1): 27–34. doi:10.1161/CIRCULATIONAHA.113.008285. PMC 4082404. PMID 24795390.
  6. Cenni V, Capanni C, Columbaro M, Ortolani M, D'Apice MR, Novelli G; et al. (2011). "Autophagic degradation of farnesylated prelamin A as a therapeutic approach to lamin-linked progeria". Eur J Histochem. 55 (4): e36. doi:10.4081/ejh.2011.e36. PMC 3284238. PMID 22297442.
  7. Cao K, Blair CD, Faddah DA, Kieckhaefer JE, Olive M, Erdos MR; et al. (2011). "Progerin and telomere dysfunction collaborate to trigger cellular senescence in normal human fibroblasts". J Clin Invest. 121 (7): 2833–44. doi:10.1172/JCI43578. PMC 3223819. PMID 21670498.
  8. Adam MP, Ardinger HH, Pagon RA, Wallace SE, Bean LJH, Stephens K; et al. (1993). "GeneReviews®". PMID 20301300.

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