Rare Medical 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 Medical News