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Diseases

Genetic and Rare Diseases Information Center (GARD)

Other Names for this Disease
  • HGPS
  • Hutchinson Gilford progeria syndrome
  • Hutchinson Gilford syndrome
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Treatment


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How might progeria be treated?

Management for progeria generally focuses on the signs and symptoms of the condition and may include the following:
  • Exercise, diet modification, and medication when the lipid profile becomes abnormal
  • Frequent small meals to maximize caloric intake
  • Oral hydration
  • Use of shoe pads for foot discomfort due to lack of body fat
  • Use of sunscreen on all exposed areas of skin
  • Nitroglycerin for angina
  • Routine anticongestive therapy if congestive heart failure is present
  • Statins for their putative effect on farnesylation inhibition
  • Anticoagulation therapy if vascular blockage, transient ischemic attacks, stroke, angina, or heart attack occur
  • Routine physical and occupational therapy to help maintain range of motion in large and small joints[1]

Although there is currently no cure for progeria, research involving treatments is ongoing and scientists have been making much progress. The results of a recently published phase II clinical trial provided preliminary evidence that lonafarnib, a farnesyltransferase inhibitor, may improve cardiovascular status, bone structure, and audiological (hearing) status in affected children.[2] A free, full-text version of this study is available on PubMed and can be viewed by clicking here.

Last updated: 11/19/2012

References
  1. Leslie B Gordon, W Ted Brown, and Francis S Collins. Hutchinson-Gilford Progeria Syndrome. GeneReviews. January 6, 2011; http://www.ncbi.nlm.nih.gov/books/NBK1121/. Accessed 11/16/2012.
  2. Leslie B. Gordon, et al. Clinical trial of a farnesyltransferase inhibitor in children with Hutchinson–Gilford progeria syndrome. Proc Natl Acad Sci. October 9, 2012; 109(41):16666–16671. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3478615/. Accessed 11/19/2012.


Management Guidelines

  • GeneReviews provides current, expert-authored, peer-reviewed, full-text articles describing the application of genetic testing to the diagnosis, management, and genetic counseling of patients with specific inherited conditions. Click on the link to view the article on this topic.

Clinical Trials & Research for this Disease

  • ClinicalTrials.gov lists trials that are studying or have studied Progeria. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.