Hutchinson-Gilford progeria syndrome (HGPS) is a rare autosomal dominant disorder estimated to affect approximately one in 20 million individuals resulting in adverse symptoms associated with premature ageing: skeletal dysplasia, joint contractures, atherosclerosis, myocardial fibrosis/dysfunction, scleroderma-like cutaneous effects, lipoatrophy, alopecia, a...
Lonafarnib is a farnesyltransferase inhibitor indicated in patients aged 12 months and older with a body surface area of at least 0.39 m to reduce the risk of mortality associated with Hutchinson-Gilford progeria syndrome (HGPS). It is also indicated in this same population for the treatment of processing-deficient progeroid laminopathies that either involve...
New Zealand Liver Transplant Unit, Auckland City Hospital, Auckland, New Zealand
Department of Gastroenterology and Hepatology, Koรง University Medical School, Istanbul, Turkey, Istanbul, Turkey
Soroka UMC, Be'er Sheva, Israel
University of Miami Schiff Center for Liver Disease, Miami, Florida, United States
Azienda Ospedaliero Universitaria di Parma, Parma, Italy
Azienda Ospedaliera Universitaria Policlinico Tor Vergata, Roma, Italy
National Institutes of Health Clinical Center, Bethesda, Maryland, United States
National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States
Ankara University Medical School, Ankara, Turkey
Ankara University Medical School, Ankara, Turkey
National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States
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