Condition: Fabry Disease · Aged 65 Years or Older · Alpha Galactosidase A Deficiency · Sponsor: Wladimir MAUHIN, Dr
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 Fabry disease remains controversial, but early initiation is recommended for long-term benefits. Despite the high cost and inconvenience of treatments, there is limited research on their efficacy in older people or on the quality of life for those aged 65 and over with Fabry disease. This study aims to assess the quality of life in this age group both with and without treatment over a period of 5 years to determine the benefits of treatment beyond the age of 65.
This description comes directly from the study's public registry record.
Djazia Bouzelmat, Clinical Research Assistant · 01 44 64 30 98 · dbouzelmat@hopital-dcss.org
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| Groupe Hospitalier Diaconesses Croix Saint-Simon | Paris, France, France | Recruiting |
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Source record: clinicaltrials.gov/study/NCT07277361