Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment
Recruiting
Observational Study
Fabry Disease
Aged 65 Years or Older
Alpha Galactosidase A Deficiency
Galactosidase A Gene Mutation
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.
At a Glance
- Age
- 65 and older
- Sex
- Any
- Study type
- Observational
- Participants needed
- 100 (estimated)
- Sponsor
- Wladimir MAUHIN, Dr · Other
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About This Trial
Fabry disease is a rare genetic disorder affecting 1 in 10,000 individuals, leading to complications such as chronic pain, heart and kidney failure, and strokes, ultimately impacting life expectancy. People with this disease are increasingly being diagnosed later in life, around the age of 65, as the condition progresses slowly with irreversible organ damage. The effectiveness of treatments for Fa…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Men and women aged 65 and over with a diagnosis of Fabry disease with, for men, a proven alpha-galactosidase A deficiency or an identified pathogenic GLA genetic variant, and for women, an identified pathogenic GLA variant.
* Minimum work-up available: ECG, 24h holterECG, card…
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