A Study of Patients With Fabry Disease (US Specific)

Recruiting Observational Study
Fabry Disease
No Placebo Group Every participant receives an active treatment — no one gets a placebo. No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Age
18 and older
Sex
Any
Study type
Observational
Participants needed
450 (estimated)
Sponsor
Amicus Therapeutics · Industry
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About This Trial

This is an observational study to evaluate the effects of treatment on long-term effectiveness, safety, and health-related quality of life (HRQOL) in patients with Fabry disease, with a main focus on migalastat.

Trial Locations
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Eligibility Criteria
I. Migalastat-treated patients (Commercial only participants) 1. Patients with Fabry disease 18 years or older with amenable GLA variants who have commenced commercial migalastat treatment within 24 months preceding enrollment, who have an eGFR greater than or equal to 30 mL/min/1.73 m2 at the time…
Contacts

Amicus Therapeutics Patient Advocacy

609-662-2000

patientadvocacy@amicusrx.com

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