Title

Rare neurological channelopathies--networks to study patients, pathogenesis and treatment

Document Type

Article

Abstract

Each of the thousands of rare neurological diseases requires a widely distributed network of centres, investigators and patients, so as to foster multidisciplinary investigations and involve sufficient numbers of patients in the discovery of disease pathogenesis and novel treatment. In this Review, we highlight the value of this collaborative approach in patient-oriented research into rare neurological channelopathies. Two networks, the Consortium for Clinical Investigations of Neurological Channelopathies (CINCH) and the Clinical Research Consortium for Studies of Cerebellar Ataxias (CRC-SCA), provide a link between patients with rare channelopathies and investigators who are studying disease pathogenesis and developing novel treatments. Interactions between patients, researchers and advocacy groups promote shared agendas that benefit patient education and recruitment, research collaboration and funding, and training and mentoring of junior investigators who are attracted to the study of the diseases that provide the focus for the two networks. Here, we discuss how linkage of national and international centres has enabled recruitment of study participants, provided opportunities for novel studies of pathogenesis, and facilitated successful clinical trials.

Medical Subject Headings

Biomedical Research (economics, methods); Channelopathies (diagnosis, epidemiology, therapy); Clinical Trials as Topic (economics, methods); Humans; Multicenter Studies as Topic (economics, methods); Patient Selection; Treatment Outcome

Publication Date

4-1-2016

Publication Title

Nature reviews. Neurology

E-ISSN

1759-4766

Volume

12

Issue

4

First Page

195

Last Page

203

PubMed ID

26943780

Digital Object Identifier (DOI)

10.1038/nrneurol.2016.18

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