The Natural History of Familial Dysautonomia
Recruiting
Observational Study
Familial Dysautonomia (Riley-Day Syndrome)
Hereditary Sensory and Autonomic Neuropathies
Hereditary Sensory and Autonomic Neuropathy 3
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
- 4 and older
- Sex
- Any
- Study type
- Observational
- Participants needed
- 400 (estimated)
- Sponsor
- NYU Langone Health · Other
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About This Trial
The study will collect clinical information from patients with FD and allow them to give blood to help develop biological markers of the disease to aid diagnosis and treatment.
This is a non-invasive, non-interventional, observation study that poses only minimal risk for participants. The study will document the clinical features of patients with FD overtime by storing their routine clinical test…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Patients of any age with a diagnosis of familial dysautonomia (FD) with molecular confirmation of the IKBKAP mutation.
* Ability to provide informed consent (or assent) and comply with the study protocol
Exclusion Criteria:
* Subjects that do not wish to be a part of the stu…
Contacts