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Cardiaccomplications occur when glycolipids build up in different heart cells; heart-related effects

worsen with age and may lead to increased risk of heart disease.High blood

pressureandcardiomyopathyare commonly observed.[citation needed]

Dermatological manifestations

Angiokeratomas(tiny, painlesspapulesthat can appear on any region of the body, but arepredominant on the thighs, around the belly button, buttocks, lower abdomen, and groin) are

common.[citation needed]

Anhidrosis(lack of sweating) is a common symptom, and less commonlyhyperhidrosis(excessive

sweating).[citation needed]

Additionally, patients can exhibitRaynaud's disease-like symptoms withneuropathy(in particular,

burning extremity pain).[citation needed]

Ocular manifestations

Cosmetic ocular involvement may be present showingcornea verticillata(also known as vortex

keratopathy), i.e. clouding of the corneas. Keratopathy may be the presenting feature inasymptomatic patients, and must be differentiated from other causes of vortex keratopathy (e.g. drug

deposition in the cornea).[7]This clouding does not affect vision.[7]

Other ocular findings can include conjunctivalaneurysms,posterior spoke-

likecataracts,papilloedema,macular edema,optic atrophy,and retinal vascular dilation.[citation needed]

Other manifestations

Fatigue,neuropathy(in particular, burning extremity pain), cerebrovascular effects leading to an

increased risk of stroke,tinnitus(ringing in the ears),vertigo,nausea, inability to gain weight,

chemical imbalances, and diarrhea are other common symptoms.[citation needed]

DiagnosisFabry disease is suspected based on the individual's clinical presentation, and can be diagnosed by

an enzyme assay (usually done onleukocytes)to measure the level ofalpha-galactosidaseactivity.

An enzyme assay is not reliable for the diagnosis of disease in females due to the random nature

ofX-inactivation.Molecular genetic analysis of the GLAgene is the most accurate method of

diagnosis in females, particularly if the mutations have already been identified in male family

members. Many disease-causing mutations have been noted. Kidney biopsy may also be suggestive

of Fabry disease if excessive lipid buildup is noted. Pediatricians, as well as internists, commonly

misdiagnose Fabry disease.[8]

TreatmentThe first treatment for Fabry's disease was approved by the US FDA on April 24, 2003. Fabrazyme

(agalsidase beta) was licensed to theGenzymeCorporation.[9]It is anenzyme replacement

therapy(ERT) designed to provide the enzyme the patient is missing as a result of a genetic

malfunction. The drug is expensive in 2012, Fabrazyme's annual cost was about US$200,000 per

patient,[10]which is unaffordable to many patients around the world without enough insurance. ERT
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is not a cure, but can allow improved metabolism and partially prevent disease progression, as well

as potentially reverse some symptoms.

The pharmaceutical companyShiremanufactures agalsidase alpha under the brand

nameReplagalas a treatment for Fabry's disease,[11]and was granted marketing approval in the EU

in 2001.[12]FDA approval was applied for the United States.[13]However, Shire withdrew their

application for approval in the United States in 2012, citing that the agency will require additionalclinical trials before approval.[14]

Pain associated with Fabry disease can be partially alleviated by ERT, but pain management

regimens may also includeanalgesics,anticonvulsants,andnonsteroidal anti-inflammatory drugs,

though the latter are usually best avoided in renal disease.

Prognosis

Life expectancywith Fabry disease for males was 58.2 years, compared with 74.7 years in the

general population, and for females 75.4 years compared with 80.0 years in the general population,

according to registry data from 2001 to 2008. The most common cause of death was cardiovascular

disease, and most of those had received kidney replacements.[15]

References

1. ^James, William D.; Berger, Timothy G.; et al. (2006). Andrews' Diseases of the Skin: clinicalDermatology. Saunders Elsevier. p. 538.ISBN0-7216-2921-0.

2. ^synd/1761atWho Named It?

3. ^Karen JK, Hale EK, Ma L (2005)."Angiokeratoma corporis diffusum (Fabrydisease)".Dermatol. Online J.11(4): 8.PMID16403380.

4. ^James, William D.; Berger, Timothy G.; et al. (2006). Andrews' Diseases of the Skin: clinicalDermatology. Saunders Elsevier. pp.

[page needed].ISBN0-7216-2921-0.

5. ^Mehta A, Ricci R, Widmer U, et al. (March 2004). "Fabry disease defined: baseline clinicalmanifestations of 366 patients in the Fabry Outcome Survey". European Journal of ClinicalInvestigation34(3): 23642.doi:10.1111/j.1365-2362.2004.01309.x.PMID15025684.

6. ^Hoffmann B, Beck M, Sunder-Plassmann G, Borsini W, Ricci R, Mehta A (2007). "Nature andprevalence of pain in Fabry disease and its response to enzyme replacement therapy--aretrospective analysis from the Fabry Outcome Survey".The Clinical Journal of Pain23(6): 53542.doi:10.1097/AJP.0b013e318074c986.PMID17575495.

7. ^abChew E, Ghosh M, McCulloch C (June 1982). "Amiodarone-induced corneaverticillata". Canadian Journal of Ophthalmology17(3): 969.PMID7116220.

8. ^Marchesoni CL, Roa N, Pardal AM, et al. (May 2010). "Misdiagnosis in Fabry disease". TheJournal of Pediatrics156(5): 82831.doi:10.1016/j.jpeds.2010.02.012.PMID20385321.

9. ^Fabrazyme Prescribing Information (USA),www.fda.gov

10. ^Genzyme Drug Shortage Leaves Users Feeling Betrayed,By ANDREW POLLACK, New York

Times, April 15, 201011. ^Keating GM (October 2012). "Agalsidase alfa: a review of its use in the management of Fabry

disease". BioDrugs26(5): 33554.doi:10.2165/11209690-000000000-00000.PMID22946754.

12. ^"Shire Submits Biologics License Application (BLA) for Replagal with the U.S. Food and DrugAdministration (FDA)".FierceBiotech.

13. ^"With A Life-Saving Medicine In Short Supply, Patients Want Patent Broken".2010-08-04.Archivedfrom the original on 14 September 2010. Retrieved 2010-09-02.

14. ^Grogan K (2012-03-15)."Shire withdraws Replagal in USA as FDA wants more trials".PharmaTimes.
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15. ^Waldek S, Patel MR, Banikazemi M, Lemay R, Lee P (November 2009). "Life expectancy andcause of death in males and females with Fabry disease: findings from the FabryRegistry". Genetics in Medicine11(11): 7906.doi:10.1097/GIM.0b013e3181bb05bb.PMID1974574
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