Table 3.

Effect of ERT in patients with FD

Organ	Background/Setting/Measures	Effect on CNS Function/Symptoms	Reference
Central nervous system
Agalsidase-α	Placebo-controlled study, 26 male patients treated for 6 mo, resting global cerebral blood flow changed by −3.48±4.57 (treated, n=14) versus +1.25±5.11 (placebo, n=12) ml/min per 100 g of tissue (P=0.03)	Improvement of cerebral blood flow	63
	Open-label study, 63 male patients treated for 18 mo, Doppler blood flow parameters (peak flow velocity, mean flow velocity, end-diastolic velocity, flow acceleration) in certain vessels improved (all P<0.01)	Improvement of cerebral blood flow	67
Agalsidase-β	Post hoc analysis of phase 4 study, 31 patients treated for 12 mo, in patients≤50 yr of age, normalized WML diameter increased significantly in placebo group (n=13, P=0.03) and remained stable in the treated group (n=18, P>0.5)	Stabilization of WMLs	68

CNS, central nervous system; WML, white matter lesion.