Farnesylation Inhibitors Allow Children with Progeria To Live Longer


Study finds progeria treatment to extend survival time by 1.6 years.

Study finds progeria treatment to extend survival time by 1.6 years.

Protein farnesylation inhibitors extend the estimated lifespan of children with progeria. That is the conclusion from a new study by Dr Leslie Gordon and colleagues published in Circulation [Epub ahead of print]. This is very exciting news for a condition known to result in premature death by the age of about 14 in most children with progeria.

Progeria is caused by a mutation in the LMNA gene that eventually leads to abnormal levels of the protein progerin that disrupt the nucleus of cells and causing children with this condition to age prematurely.

Children with progeria begin to display many characteristics of accelerated aging at around 18-24 months of age. Progeria signs include growth failure, loss of body fat and hair, aged-looking skin, stiffness of joints, hip dislocation, generalized atherosclerosis, cardiovascular (heart) disease and stroke. Almost all children with progeria die of atherosclerosis (heart disease) at an average age of fourteen years.

Currently there is no approved treatment for progeria. However, since 2007, protein farnesylation inhibitors, such as lonafarnib, have been given to some patients to reduce the toxic effects of progerin in single arm studies. Now that about 6 years has passed since lonafarnib was first administered in a clinical trial to children with progeria, enough time has elapse to allow for a survival analysis to take place.

Click here to read the full article on Rare Disease Report.

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