Authors:
Leonard H, Anderson A, Bebbington A, Bao X, Wong K, Williams S, Downs J
Authors notes:
Developmental Medicine and Child Neurology. 2013:online
Keywords:
Rare disorders InterRett database Rett syndrome innovative
Abstract:
Our investigation used the infrastructure of InterRett, established in 2002 with dual aims of encour- aging international collaboration and ascertaining the large case numbers required for statistically meaningful research in Rett syndrome.
InterRett was ahead of its time in its global approach to rare disease research including the use of the internet for data collection.
An international approach involving standardization of data collection methods for both phenotype and genetic information is increasingly recognized tobe pivotal in rare and complex disease research, and InterRett will have a role to play.
We therefore believe that a further important question for the Rett syndrome research community is: how do we work collaboratively with families to develop innovative approaches to improved understanding and management in Rett syndrome?