Hutchinson-gilford progeria syndrome

Clinical Research/Studies

Are there clinical trials for progeria?

In 2012, the first-ever drug treatment developed for progeria began clinical trials. The drug is called lonafarnib, and early studies have shown promise that this drug may be able to extend the life span of patients with progeria. The studies are still ongoing. If you would like to get involved in this or other research, reach out to the Progeria Research Foundation at http://www.progeriaresearch.org/index.html. They are the main, centralized organization driving clinical research and advocacy for this rare disorder. As of July 2015, lonafarnib is available on a research basis to progeria patients. Please visit http://www.progeriaresearch.org/lonafarnib-pre-clinical-program.html for more information on the pre-clinical program.

SOURCE: Emory University - Department of Human Genetics in collaboration with ThinkGenetic • https://www.thinkgenetic.com/diseases/hutchinson-gilford-progeria-syndrome/clinical-research/6112 • DATE UPDATED: 2016-06-16

References

Progeria Research Foundation

http://www.ncbi.nlm.nih.gov/books/NBK1121/

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