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BURDEN OF DISEASE

Rare diseases

Lysosomal storage disorders

Fabry disease

• Classical Fabry disease
• Late-onset Fabry disease
• Classical and late-onset Fabry disease: a comparison

Genetic inheritance of Fabry disease

Epidemiology of Fabry disease

Impact of Fabry disease

DIFFERENTIAL DIAGNOSIS

Signs and symptoms of Fabry disease

Misdiagnosis of Fabry disease

Getting the diagnosis right

Diagnosis of Fabry disease

• Disease severity indexes
• Genetic testing
• Alpha-galactosidase A
• Globotriaosylsphingosine
• Clinical indicators
• Family screening
• Prenatal and newborn screening

DISEASE MANAGEMENT

Patient-centric disease management

• Management of Fabry disease
• Support and counselling for Fabry disease

Symptomatic management of Fabry disease

Enzyme replacement therapy

• Agalsidase alfa
• Agalsidase beta

Chaperone therapy

• Migalastat

MEETINGS & EVENTS

WORLDSymposium 2021

SSIEM 2020

Key congress calendar 2022

PUBLICATIONS & EXPERT INSIGHTS

TAKEDA'S COMMITMENT TO RARE DISEASES

FABRY DISEASE RESOURCES BY TAKEDA

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