Enzyme Replacement Therapy Stabilized White Matter Lesion Progression in Fabry Disease | Zendy

Andreas Fellgiebel | Zendy; M Gartenschläger | Zendy; Kerstin Wildberger | Zendy; Armin Scheurich | Zendy; Robert J. Desnick | Zendy; Katherine B. Sims | Zendy

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Enzyme Replacement Therapy Stabilized White Matter Lesion Progression in Fabry Disease

Author(s) -

Andreas Fellgiebel,

M Gartenschläger,

Kerstin Wildberger,

Armin Scheurich,

Robert J. Desnick,

Katherine B. Sims

Publication year - 2014

Publication title -

cerebrovascular diseases

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 1.221

H-Index - 104

eISSN - 1421-9786

pISSN - 1015-9770

DOI - 10.1159/000369293

Subject(s) - medicine , enzyme replacement therapy , fabry disease , hyperintensity , placebo , stroke (engine) , left ventricular hypertrophy , cardiology , magnetic resonance imaging , population , surrogate endpoint , creatinine , pathology , radiology , disease , blood pressure , mechanical engineering , alternative medicine , environmental health , engineering

The central nervous system manifestations in Fabry disease (FD) include progressive white matter lesions (WMLs) and stroke. Due to progressive microvascular involvement, men and women with FD over 35 years of age develop WMLs. Moreover, the prevalence of stroke has been estimated to be 12 times higher in FD compared with the general population. Enzyme replacement therapy (ERT) is available and has shown beneficial effects on renal, cardiac, and peripheral nerve function in FD, but the ERT effect on the progression of WMLs, or the reduction in cerebrovascular events, remains unknown.

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