Hereditary Neuropathy Foundation

Conclusion

Hereditary Neuropathy Foundation meets the 20 Standards for Charity Accountability.

Purpose

Stated Purpose:
Hereditary Neuropathy Foundation (HNF) is a non-profit 501(c)3 organization which mission is to increase awareness and accurate diagnosis of Charcot-Marie-Tooth (CMT) and related inherited neuropathies, support people living with CMT and their families with critical information to improve quality of life, and fund research that will lead to treatments and cures.

Year, State Incorporated:
2001, NY

Programs

HNF’s patient centered programs are supportive of patients, caregivers, health care providers and others (biotech, pharma, and regulatory agencies) to develop therapies for Charcot-Marie-Tooth (CMT) and related inherited neuropathies (IN).  
CMT is a group of inherited neurological disorders that primarily affect the peripheral nerves. These peripheral nerves are responsible for transmitting signals between the brain and spinal cord to the muscles and sensory organs throughout the body. The most common symptoms include muscle weakness, muscle atrophy (shrinkage), and sensory loss. People with CMT often experience difficulties with muscle coordination, balance, and walking. In some cases, the disease can affect the muscles of the hands, leading to issues with fine motor skills. 
HNF’s Therapeutic Research in Accelerated Discovery (TRIAD) is a collaborative effort of  academia, government, and industry to develop treatments for CMT. TRIAD involves many groups that span the drug discovery, drug development, and diagnostics continuum. HNF raises funds to support the development of therapies for many CMT subtypes. The innovative research projects include the development of cell and animal models, and biomarker development. Projects are funded based upon their likelihood of success and include FDA repurposed drugs, novel drugs, and gene therapies. HNF is largely focused on clinical trial readiness to ensure success. Currently, HNF has 30 designated CMT Center of Excellence (6 are pediatric sites) that demonstrate strengths in providing excellence in clinical care and most of them participate in CMT clinical trials. HNF continues its regulatory initiatives by building upon the HNF hosted Externally-led Patient Focused Drug Development (PFDD) meeting with FDA in 2018 and Critical Path Innovation Meeting (CPIM) with a focus on incorporating decentralized  and video capture for upcoming clinical trials. 
As part of TRIAD, the Global Registry for Inherited Neuropathies (GRIN) was established as an IRB-approved patient registry and research consortium to conduct  natural history studies for all CMT subtypes. GRIN is a user-friendly online platform allowing patients, caregivers, clinicians, and researchers the ability to manage health data to elevate patient care, advance research, support clinical trial designs and enhance therapy development with TRIAD partners. The data collected is stored, analyzed and published by HNF staff with the oversight and input of TRIAD Council experts. Data includes patient-reported details, curated genetic report uploads and results of clinical CMT validated scales. The data has been instrumental in identifying the burden, diagnostic journey and prevalence of CMT. 
HNF continues to expand its resources to support the CMT community with a focus on enhancing clinical trial endpoints and outcomes. In 2021, HNF conducted an innovative remote data collection   pilot study utilizing the power of computer learning by developing the CMT Mobile app. The CMT Mobile App empowered the patient to collect prescribed photos and videos in their own home to precisely capture the challenges with activities of daily living (disease burden) due to CMT. This study has helped to better identify disease progression and/or improvement of CMT characteristics and symptoms. 
In 2023, HNF partnered with CombinedBrain to launch the first-ever CMT Biobank, part of the GRIN consortium. GRIN registrants have the opportunity to participate in translational research by providing samples which include blood, tissue, skin fibroblasts, Induced Pluripotent Stem Cells (iPSCs), and more.  All samples are made available to the research community and industry to support the development and validation of biomarkers in CMT, an essential regulatory (FDA and EMA) requirement for clinical trials. 
HNF’s CMT Genie, is a patient-initiated genetic testing program to support genetic diagnosis by offering patients virtual genetic counseling, with an option to obtain a prescription for an at-home test, with a qualified genetic counselor to seek a genetic diagnosis. The CMT Genie program has supported hundreds of patients who have then agreed to upload their reports into GRIN. 
To financially support programs, HNF relies on Team CMT. Team CMT helps to increase awareness and raise funds. The fundraising platform is personalized for each participant to participate in events globally. 
The patient voice is at the core of HNF’s mission and in partnership with Inspire™ offers patients access to a robust and interactive online CMT social community with over 10,000 members. 
HNF’s Movement is Medicine™ (MiM) program continues to offer an adaptive fitness program for free to encourage and support physical activity and a healthy lifestyle for those people affected by CMT and other debilitating diseases. Initially launched as in person summits, quickly became virtual during COVID and continues to thrive by offering virtual classes weekly with Fitness Ambassadors.  

For the year ended June 30, 2022, Hereditary Neuropathy Foundation's program expenses were:

Governance & Staff

Chair's Profession / Business Affiliation
Not disclosed

Board Size
14

Paid Staff Size
7

Fundraising

Method(s) Used:
Invitations to fund raising events, Print advertisements (newspapers, magazines, etc.), Grant proposals, Internet, Appeals via Social Media (Facebook, etc.)

Tax Status

This organization is tax-exempt under section 501(c)(3) of the Internal Revenue Code. It is eligible to receive contributions deductible as charitable donations for federal income tax purposes.

Financial

The following information is based on Hereditary Neuropathy Foundation's audited financial statements for the year ended June 30, 2022.

Source of Funds

Programs: 88% Fundraising: 7% Administrative: 5%