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Progeria

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A rare genetic condition that produces rapid aging in children.

Symptoms strongly resemble normal human aging, but occur in young children.

It usually is not passed down through families.

Autosomal dominant premature aging disorder.

In more than 90% of cases a mutation of a cytosine with thymine at position 1824 in LMNA, which encodes lamin A.

Rarely seen in more than one child in a family.

Symptoms:

Growth failure during the first year of life

Narrow, shrunken or wrinkled face

Baldness

Severe failure to thrive

Loss of eyebrows and eyelashes, and scalp hair

Short stature

Macrocephaly

Open fontanelle

Micrognathia

Dry, scaly, thin skin

Limited range of motion

Joint contracture, skeletal dysplasia

Teeth – delayed or absent

Laboratory tests may show insulin resistance

Skin changes similar to that seen in scleroderma

Loss of subcutaneous fat, and vascular smooth muscle cells

Generally normal cholesterol and triglyceride levels

Cardiac stress testing may reveal signs of early atherosclerosis of blood vessels.

Genetic testing reveals changes in the gene (LMNA) that causes progeria.

A genetic condition that occurs as a new mutation, and is rarely inherited, as carriers usually do not live to reproduce.

No specific treatment for progeria, but aspirin and statin medications may be used to protect against a heart attack or stroke.

Causes early death with average lifespan 13 years.

The cause of death is usually related to the heart or a stroke.

There is no cure.

Lonafarnib marketed as Zokinvy, prevents the accumulation of a structural proteins as defective progeria.
 This protein buildup accelerates cardiovascular disease and children with progeria do not survive past the age of 13 years as a result.
After the first three years of treatment patients on this drug lived in average of three months longer than untreated patients, and after 11 years follow-up treated patients lived an average of 2.5 years longer than untreated patients.
Most common adverse reactions with Lonafarnib were vomiting, diarrhea, infection, nausea, decreased appetite, and fatigue.
 
DNA editing with CRISPR-Cas9 is under investigation.

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