Hereditary amyloidosis, transthyretin-related
Clinical trials for Hereditary amyloidosis, transthyretin-related explained in plain language.
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Can a new questionnaire capture life with hereditary amyloidosis?
Knowledge-focused CancelledResearchers in Argentina are developing and testing a self-administered questionnaire to measure how hereditary transthyretin amyloidosis affects patients' physical, emotional, and social quality of life. The study includes input from neurologists, a cardiologist, a psychologist,…
Matched conditions: HEREDITARY AMYLOIDOSIS, TRANSTHYRETIN-RELATED
Sponsor: Hospital General de Niños Pedro de Elizalde • Aim: Knowledge-focused
Last updated Sep 19, 2026 00:00 UTC
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Blood marker may predict nerve damage onset in rare genetic disease
Knowledge-focused OngoingThis observational study is looking at a protein called neurofilament light chain (NfL) in the blood of people who carry a gene variant for hereditary ATTR amyloidosis, both those without symptoms and those with nerve damage. Researchers want to see if NfL levels can help detect …
Matched conditions: HEREDITARY AMYLOIDOSIS, TRANSTHYRETIN-RELATED
Sponsor: Alnylam Pharmaceuticals • Aim: Knowledge-focused
Last updated Aug 14, 2026 00:00 UTC