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When to treat

Think Fabry, think early treatment to help slow or prevent life-threatening disease progression.1,2

Fabry disease is classified into two main phenotypes:2,4

  • Classic – absent or very low α-GAL A activity, multiple-organ systems involved, presentation generally begins in childhood
  • Nonclassic – also referred to as late-onset, varying levels of residual α-GAL A activity and symptoms are more variable, most frequently beginning in adulthood

Considerations for initiating ERT in each of the following:3

person

ERT should be considered and is appropriate in adult males with classic Fabry disease (symptomatic or asymptomatic)

Treatment decisions may be influenced by2:

  • Advanced, elderly age of the patient
  • Severe comorbidity
person

ERT should be considered in adult classic females upon early signs of renal, cardiac, CNS complications, pain, and GI issues2,5

Signs/symptoms suggesting major organ involvement2,5

Kidney_Icon

Proteinuria/albuminuria not attributable to other causes, evidence of renal impairment

Heart_Icon

Symptomatic cardiac disease not due to other causes (dyspnea, palpitation, syncope, chest pain)

Brain_Icon

Stroke or TIA

Pain_Icon

Neuropathic pain, pain crises, Fabry disease neuropathy

Stomach_Icon

Neuropathic pain, pain crises, Fabry disease neuropathy

Sweating_Icon

Exercise intolerance and impaired sweating

person

Lab/histological/imaging evidence of injury to kidney, heart, or CNS2,5

Kidney_Icon

GFR <90mL/min/1.73m2, adjusted for age >40 years, persistent albuminuria >30 mg/g, podocyte foot process effacement on renal biopsy
 Moderate or severe GL-3 inclusions in a range of renal cell types

Heart_Icon

Asymptomatic cardiac disease (cardiomyopathy or arrhythmia, cardiac fibrosis on contrast cardiac MRI)

Brain_Icon.png

Silent strokes, cerebral white matter
lesions (on brain MRI)

person

Lab/histological/imaging evidence of injury to kidney, heart, or CNS2,5

Kidney_Icon

GFR <90mL/min/1.73m2, adjusted for age >40 years, persistent albuminuria >30 mg/g, podocyte foot process effacement on renal biopsy
 Moderate or severe GL-3 inclusions in a range of renal cell types

Heart_Icon

Asymptomatic cardiac disease (cardiomyopathy or arrhythmia, cardiac fibrosis on contrast cardiac MRI)

Brain_Icon.png

Silent strokes, cerebral white matter
lesions (on brain MRI)

Safety

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MAT-BE-2500570 (ver. 1) 05/2025