Progeria clinical trial at Boston Children’s Hospital

Progeria clinical trial at Boston Children’s Hospital

“Progeria” isn’t just one disease, but a group of rare, fatal genetic conditions that give children the appearance of aging prematurely (the name, which comes from the Greek, means “prematurely old”). The classic type of progeria, which we’ll focus on in these pages, is Hutchinson-Gilford Progeria Syndrome (HGPS), named for the two doctors who first described it in England in the late 1800s.

Although children with progeria (pronounced “pro-JEER-ee-a”) are born looking healthy, they begin to show many features of premature aging at around 18 to 24 months of age. They have a physical appearance that is very similar from child to child, even when they come from different ethnic backgrounds.

Children with progeria have normal intelligence, and think and act just like other children their age. They can generally attend school, participate in sports and play with other children.

As children with progeria get older, they start to get osteoporosis and atherosclerosis (hardening of the arteries), and may suffer heart attacks and strokes at as young as5 years old. They die, usually from heart disease, at an average age of 13.

Learn more:

Progeria cell images:

Capell BC; et al. Inhibiting farnesylation of progerin prevents the characteristic nuclear blebbing of Hutchinson-Gilford progeria syndrome. Proc Natl Acad Sci U S A 2005 Sep 6; 102(36):12879-84. (Copyright 2005 National Academy of Sciences, U.S.A.)

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