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Fabry Disease Mutation Analysis by Sequencing

Fabry disease is an X-linked disorder of glycosphingolipid catabolism caused by deficient activity of the lysosomal enzyme, α-galactosidase A (GLA). Signs and symptoms include angiokeratomas, ocular opacities, painful neuropathy in the extremities, progressive renal insufficiency and cardiac hypertrophy. Untreated, death usually occurs in early to mid-adulthood. Treatment is available for individuals with Fabry disease.

Confirmation of the clinical diagnosis in affected hemizygous males can be accomplished by demonstration of deficient α-galactosidase A activity in plasma or leukocytes. However, determination of carrier status in heterozygous females typically cannot be accomplished by testing α-galactosidase A activity.

Indications for Fabry disease mutation analysis by sequencing include: family history of Fabry disease, clinical characteristics of Fabry disease, GLA deficiency by enzyme analysis, positive newborn screen for Fabry disease, or prenatal testing for at-risk fetuses.

Full Sequencing:
83891(1), 83892(8), 83898(8), 83904(16), 83909(16), 83912(1)

Single Exon Sequencing:
83891(1), 83892(1), 83898(1), 83904(2), 83909(2), 83912(1)

Each additional exon:
83898(1), 83892(1), 83909(2), 83904(2)


Specimen Requirements:
  1. Blood:
    • ACD-A (yellow-top) or EDTA (lavender-top) tubes.
    • Adult: 10 ml whole blood.
  2. Bloodspot:
    • Guthrie cards (dried blood spot): Ensure sufficient quantity of blood to saturate and completely fill a minimum of three preprinted circles on the paper.
  3. Prenatal:
    • All prenatal specimens (including cord blood) must be accompanied by samples from both parents.
    • Discard first 2 ml; then 15 ml amniotic fluid in 15 ml orange-top polypropylene tube or 10-15 mg chorionic villi in laboratory-provided screw-top tubes with sterile transport medium or two T-25 flasks of confluent fetal cells.
    • Additional sample must be obtained for back-up culture at one of our cytogenetics laboratories or another facility.
    • Partial Fabry gene sequencing may be performed using direct chorionic villi samples, but cultured cells are preferred.
    • Full Fabry gene sequencing requires cultured cells from amniotic fluid or chorionic villi samples.

Turnaround Time:

21 days


Client Services: Reproductive/Genetics   800-848-4436

Specimen Services/Pickup:  877-246-1226

Reproductive/Genetics Client Services

Tel: 800-848-4436