In general, the collection, analysis and dissemination of data on disease progression and patient responses to long-term disease management strategies in registries represents a valuable way to improve understanding of the disease and keep medical professionals up to date on the latest advances. Not all patients have access to CMT centers or medical professionals trained to recognize disease, therefore limiting the potential to gather natural history information on a multitude of the population and capture different groups and geographic regions that include age, co-morbidities, current palliative therapies are necessary in a grossly undiagnosed and misdiagnosed community.
The Hereditary Neuropathy Foundation & Hannah’s Hope Fund launched the Global Registry for Inherited Neuropathies (GRIN) with the primary objective to identify patients with inherited neuropathies to get a better understanding of disease phenotypes, which vary among all the different types, and sub-types of CMT and related inherited neuropathies. With GRIN, patients self-register on the internet. GRIN includes a comprehensive series of questions covering many areas and a sample of these are now provided for: diagnosis / assessments, genetic test results, general health, neurology, orthopedic, alternative / complimentary therapies, skin / hair, clinical trials and research / biorepository, registry.
This registry is therefore complementary to and far more extensive than a contact registry such as that managed by The Inherited Neuropathy Consortium.
GRIN covers all inherited neuropathies and is managed by PatientCrossroads which is a professional organization that has developed registries for more than 250 diseases. These are used by participants in more than 75 countries.
GRIN can be used to connect patients, healthcare providers, researchers, clinical investigators, regulators, policy-makers, industry and beyond. Establishing GRIN addresses three critical needs. First, scientists studying various forms of inherited neuropathy need accurate information to understand the effects of specific forms of neuropathy. Second, scientists who are conducting preclinical studies may need tissue samples from registrants who agreed to be contacted by GRIN if samples were needed to advance research for their form of inherited neuropathy. Additionally, clinicians, pharmaceutical and biotechnology companies ready to begin trials will need the GRIN administrator to distribute and advertise the study and clinicaltrials.gov study link. If a patient looks like a good match for a scientist’s research the scientist will contact HNF. The Foundation or the foundation’s agent will then contact the inherited neuropathy family. Scientists will not contact families directly and will only be given de-identified patient data.
Another critical component of GRIN is to get a better understanding of disease progression in the general population of both diagnosed and undiagnosed patients. Environmental factors and contraindicated drugs have been confirmed to exacerbate CMT and/or present onset of symptoms in asymptomatic patients. This obviously can alter disease progression and may be relevant to the natural history and clinical outcomes of treatments.
GRIN represents a great opportunity for inherited neuropathy patients to help themselves.