Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Recruiting Observational Study
Inherited Disease Rare Diseases Metabolic Disease Undiagnosed Disease Neurologic Disorder Neuro-Degenerative 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
100 (estimated)
Sponsor
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta · Other
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About This Trial

General aim of the study is the improvement of the clinical knowledge of ultra-rare inherited metabolic and degenerative neurological diseases (prevalence less than 5:100,000) in adulthood through the systematic longitudinal collection of clinical, laboratory and instrumental data.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Age \>= 18 years * Subjects with ultra-rare inherited degenerative and metabolic neurological diseases * Subjects with undiagnosed neurological diseases (when supposed to be inherited) Exclusion Criteria: * none
Contacts

Ettore Salsano, MD

+39022394

ettore.salsano@istituto-besta.it

CONTACT

Renato Mantegazza, MD

+39022394

crc@istituto-besta.it

CONTACT