Table 3. Positive effects reported for enzyme replacement therapy (ERT) in Fabry’s disease.

Organ (system)	Symptoms	Effect with enzyme replacement therapy	Study design	Source
Nervous system	Pain	Pain reduction	Double blind, randomized, controlled	23
			Cohort study	3
	Peripheral neuropathy	Improved function of peripheral nerves	Open controlled study	e45
		Improved function of peripheral nerves	Open extension study of a double blind, randomized, controlled trial	e46
	Increased regional blood circulation in the CNS	Reversible	Double blind, randomized, controlled + 12 months open extension study	e47
Gastrointestinal tract	Abdominal pain	Reduction	Cohort study	4
	Diarrhea, constipation	Tendency toward normalization of bowel movement	Cohort study	4
Skin	Sweating	Normalization of sweating	Open extension study of a double blind, randomized, controlled trial	e46
Ear	Hearing impairment, hearing loss	No progression Improvement in less severe hearing loss	Open controlled study	8
			Double blind, randomized, controlled	19
	Disturbances of equilibrium	Regression	Open controlled study	8
Kidneys	Renal failure	Gb3 storage in glomeruli dissolved	Double blind, randomized, controlled	22
		Creatinine clearance improved; decrease in number of abnormal glomeruli	Double blind, randomized, controlled	23
		Fall in GFR is stopped	Open controlled study	e41
			Cohort study	e42
		Creatinine values fall	Cohort study	25
Heart	Cardiomyopathy	Regression	Double blind, randomized, controlled	20
		No progression	Cohort study	e43
		Regression	Open controlled study	e44
	Disrupted variability of cardiac frequency	Normalization	Open controlled study	21
	Angina pectoris, myocardial infarction	Gb3 storage in endothelial cells dissolved	Double blind, randomized, controlled	22
Quality of life		Improvement	Cohort study	25