Rare Endocrinology News
5 Facts you should know about
Fabry disease
1
Symptoms include muscle weakness, respiratory failure, and cardiomyopathy
2
Symptoms are typically first experienced in early childhood and can be very difficult to diagnose; the rarity of Fabry disease to many clinicians sometimes leads to misdiagnoses
3
Age of onset ranges from infancy to adulthood
4
Respiratory and cardiac failure are leading causes of mortality
5
The majority of patients on current standard of care decline after ~2 years on standard therapy
Rare Endocrinology News
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