Fabry disease

From WikiMD.org
Jump to navigation Jump to search

Fabry disease (pronounced: /ˈfæbri/) is a rare genetic disorder, which is often inherited in an X-linked recessive manner. It is named after the dermatologist Johannes Fabry, who first described the condition in 1898.

Etymology

The disease is named after Johannes Fabry, a German dermatologist who first described the condition in 1898. The term "Fabry" is derived from the Latin "faber," meaning "smith" or "craftsman."

Definition

Fabry disease is a type of lysosomal storage disorder caused by a deficiency of the enzyme alpha-galactosidase A. This deficiency leads to the accumulation of a type of fat called globotriaosylceramide (Gb3) in the body's cells, causing a range of symptoms including pain, kidney dysfunction, heart disease, and stroke.

Symptoms

The symptoms of Fabry disease can vary widely among affected individuals. They may include episodes of pain, particularly in the hands and feet (acroparesthesia); clusters of small, dark red spots on the skin (angiokeratoma); a decreased ability to sweat (hypohidrosis); cloudiness of the front part of the eye (corneal opacity); problems with the gastrointestinal system; ringing in the ears (tinnitus); and hearing loss.

Diagnosis

Diagnosis of Fabry disease is often made through a blood test to measure the level of alpha-galactosidase A enzyme activity. Genetic testing can also be used to identify mutations in the GLA gene, which causes Fabry disease.

Treatment

Treatment for Fabry disease often involves enzyme replacement therapy (ERT) to replace the missing or deficient alpha-galactosidase A enzyme. Other treatments may include pain management, kidney dialysis or transplantation, and treatments for heart disease.

See also

References

External links

Esculaap.svg

This WikiMD article is a stub. You can help make it a full article.


Languages: - East Asian 中文, 日本, 한국어, South Asian हिन्दी, Urdu, বাংলা, తెలుగు, தமிழ், ಕನ್ನಡ,
Southeast Asian Indonesian, Vietnamese, Thai, မြန်မာဘာသာ, European español, Deutsch, français, русский, português do Brasil, Italian, polski