HNF is pleased to announce that the Orphan Disease Center (ODC) has awarded a Million Dollar Bike Ride (MDBR) 2020 CMT grant to Florian P. Thomas, MD, MA, PhD, MSc, Director of the Hereditary Neuropathy Center at Hackensack University Medical Center and Founding Chair and Professor of the Department of Neurology at Hackensack Meridian School of Medicine, along with his team at Hackensack Meridian Health.

This marks HNF’s second consecutive year participating in the annual MDBR to advance research for rare diseases. The 2020 event was held virtually, and HNF surpassed its $25,000 fundraising goal by raising $27,545. The ODC matched that amount, bringing the total grant award to $55,090.

About the Grant

The funded project is titled: Global Registry for Inherited Neuropathies (GRIN) Natural History Study Data Assessment of Charcot-Marie-Tooth (CMT) Patients to Support Virtual Clinical Studies.

The project will evaluate years of patient-reported data collected through the GRIN Registry and correlate findings to specific patient groups defined by genotype (genetic subtype), phenotype (observable characteristics), and clinical features. The results will strengthen HNF’s virtual natural history studies, validate outcome measures, help identify potential biomarkers, and support therapeutic research and clinical trials.

Specific Aims

  • Evaluate GRIN data to uncover previously unknown connections between genetic subtypes and clinical presentation in CMT patients.
  • Determine whether there are additional early signs of CMT that should be targeted for further research and treatment development.
  • Improve diagnostic accuracy and timing so that interventions can be applied earlier, when they may have greater impact.
  • Share findings through peer-reviewed publications.

To learn more about HNF’s research programs or to join the GRIN Registry, visit our research page.