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Clinical Study

Modify OLE

The purpose of the study is to evaluate if the long-term intake of the new drug, called lucerastat, is safe and well-tolerated in adults with Fabry disease.You have been asked to take part because you have Fabry disease and have completed the MODIFY study. Fabry disease is a rare, inherited disease caused by a genetic fault (mutation) which leads to a harmful accumulation of fatty substances in the body. This can cause symptoms of pain such as a burning feeling, shocks or shooting pain, tingling, pins, and needles, stabbing or numbness in hands and feet. It can also cause stomach symptoms like diarrhea and stomach pain. Fabry disease can also cause damage to vital organs like the heart, kidney, and brain.


For more information contact:

Jenny Billy

IRB#: IRB_00137274 | PI: Nicola Longo | Department: PEDIATRIC GENETICS | Approval Date: 2020-12-02 07:00:00
Specialties: Pediatric Genetics

Who can participate?

 Gender: All

 Age: Under 7 or over 18 years old

 Volunteers: Volunteers with special conditions

 Location: In Person

Will I be paid for my time?


Last Updated: 4/5/21