Patient-Powered Registry Boosts the Study of Charcot-Marie-Tooth Disease and Collects Data for Clinician Observation
NEW YORK, Sept. 3, 2021 /PRNewswire/ -- The Hereditary Neuropathy Foundation (HNF) is leading the charge in providing the research community the critical data, both patient-reported and clinician-observed, to boost the study of Charcot-Marie-Tooth (CMT) disease. HNF's Global Registry for Inherited Neuropathies (GRIN), an IRB-approved, online database collects genetic reports, and specific diagnostic and medical tests, as well as self-reported patient data through the use of surveys. Additionally, visual documentation of challenges with activities of daily living, and expert assessment of the progression of the disease is collected. By enrolling patients in GRIN, HNF hopes to provide insights for clinical trial design, more rapid patient enrollment in clinical trials, access to patients without geographic boundaries, overcome language barriers, support inclusion and diversity, and much more.
Natural history studies are critical to understanding conditions such as Charcot-Marie-Tooth (CMT) and advancing research to discover and develop new therapies. GRIN's centralized, virtual natural history study platform allows patients to self-enroll and regularly update their information, and it can also be accessed and evaluated by the healthcare provider and then de-identified to share with investigators which will be extremely valuable to all stakeholders and expeditiously lead to new therapies.
HNF identified several gaps in existing CMT natural history studies: the lack of collaboration amongst stakeholders, the lack of clinician observation of clinically relevant data, and the need to enroll more CMT patients. In response, HNF has enhanced the value of GRIN by developing an innovative mobile phone app, the "CMT Medical Journal", to precisely capture the daily challenges of living with CMT. Prescribed photos and videos based upon validated scales such as Overall Neuropathy Limitation Score (ONLS) and CMTPedS, that would typically be observed by the healthcare provider in a clinic visit, are captured with CaptureProof's Smart Medical Camera™ in the mobile app. Ultimately, AI will be utilized to provide objective analysis. The mobile app is currently only available for iPhone. It's a HIPAA-secure platform that allows the patient to share their data with their healthcare professionals. By decentralizing the natural history study data collection, more patients can be enrolled.
From Allison Moore, HNF's Founder and CEO, "I am so excited to break the barriers by collecting high-quality patient data that is both patient-powered and clinician-observed to support industry and FDA to accelerate therapies for CMT which may be a model for other rare diseases with unmet medical needs."
With the addition of the "CMT Medical Journal" to the GRIN platform we enable more robust data collection with insights that are both patient-reported and clinician-observed. This two arm approach will shed more insights on CMT research by comparing the data, including genetic reports.
"Our goals are to enhance data-sharing capability amongst stakeholders by supporting translational research and to help our industry partners develop well-designed clinical trials, accelerate patient recruitment and provide robust data-sets to share with regulators", says HNF Advocacy Director, Joy Aldrich.
HNF needs all CMT patients to create a profile and complete the GRIN surveys, and if eligible, they may be invited to enroll in the "CMT Medical Journal" pilot study. Please visit https://www.hnf-cure.org/registry/ today to get started.
About Hereditary Neuropathy Foundation (HNF)
HNF is a non-profit 501(c) 3 organization whose mission is to increase awareness and accurate diagnosis of CMT and related inherited neuropathies, support patients and families with critical information to improve quality of life, and fund research that will lead to treatments and cures. HNF developed the Therapeutic Research in Accelerated Discovery (TRIAD) program, a collaborative effort with academia, government and industry, to develop treatments for CMT. Currently, TRIAD involves many groups that span the drug discovery, drug development and diagnostics continuum.
Allison Moore
646-429-0981
[email protected]
Website: www.hnf-cure.org
SOURCE Hereditary Neuropathy Foundation
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