THE NEUROMUSCULAR DISEASE FOUNDATION

What THE NEUROMUSCULAR DISEASE FOUNDATION does

The Neuromuscular Disease Foundation (NDF), formerly the HIBM Foundation, is a 501(c)(3) nonprofit charitable organization founded in 2010 and headquartered at 269 S. Beverly Drive, Suite 1206, Beverly Hills, California. NDF is purpose-built around GNE Myopathy (GNEM/HIBM), a rare, debilitating muscular dystrophy with no approved treatment or cure, and operates the International Gene Therapy Development Program (IGTDP) as its flagship research initiative to develop a gene therapy for GNEM and advance it through FDA-approved clinical trials in partnership with Charles River Labs. The foundation has cumulatively funded approximately $9M-$10M in GNEM research and programs and supports a network of 43 associated scientific advisors.

NDF's product surface comprises research initiatives (IGTDP, current NDF-funded studies), patient programs (GNEM Multidisciplinary Clinics, Patient & Carrier Advocacy, Genetic Screening), and education assets (Learning Library with Speaker Series, podcasts, webinars, and patient stories). Patient support is delivered through Certified Patient Advocates in 50+ countries, and the organization served 4,873 people annually with a base of 5,000 individual supporters. Technical execution is outsourced to specialized partners (Charles River Labs for drug development, NHGRI at NIH, and academic investigators at Yale, Hadassah Hebrew University Medical Center, Nationwide Children's Hospital, and others).

NDF generates revenue exclusively through donations and charitable contributions, including individual giving, monthly giving programs, corporate partnerships, event fundraising, and grants (e.g., $2.5M challenge grant in 2018, $700K in 2019, $750K+ in 2021). The organization is governed by a Board of Directors (expanded in February 2023) and led by Executive Director Geoffrey M. Gee, Esq. (appointed March 2022), succeeding Lalé Welsh (2015-2022) and founder Carolyn Yashari Becher (2010-2015). It has no parent company and no venture capital or private equity ownership.

Name

THE NEUROMUSCULAR DISEASE FOUNDATION

Legal name

The Neuromuscular Disease Foundation

Website

https://curegnem.org

Company type

Private

Founded year

2010

Operating status

Operating

Headcount range

1–10 employees

Short description

The Neuromuscular Disease Foundation is a 501(c)(3) nonprofit founded in 2010 that funds gene therapy research through its IGTDP program and delivers patient advocacy, education, and genetic screening for individuals with GNE Myopathy across 50+ countries.

Ownership category

akta.pro rank

Product category

Rare Disease Patient Advocacy and Research

NAICS

Voluntary Health Organizations (813212), Research and Development in Biotechnology (except Nanobiotechnology) (541714), Social Assistance (624), Scientific Research and Development Services (5417)

SIC

Services-Social Services (8300), Services-Health Services (8000), Services-Misc Health & Allied Services, Nec (8090), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS) (BPAGACAA)

akta.pro secondary industries

Neurogenetics & Rare Neurologic Diseases (HLAKAIAO), Neuromuscular Medicine (ALS, Myasthenia, Neuropathy) (HLAKAIAD), Global Health Research, Evidence & Technical Assistance Organizations (HLAJAKAN)

Keywords

• Rare disease research
• Patient advocacy services
• Gene therapy development
• Neuromuscular disease support
• Nonprofit foundation programs

Headquarters

HQ city

Beverly Hills

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

Personnel, Operations, Marketing or Sales, Others, Technology or R&D

Revenue model

1. Donations and Fundraising: NDF is a 501(c)(3) nonprofit organization that generates revenue through individual donations, monthly giving programs, corporate partnerships, and event fundraising. The foundation relies on charitable contributions to fund research programs and patient advocacy initiatives.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels6 records

Core offering

The Neuromuscular Disease Foundation is a 501(c)(3) nonprofit foundation that funds research into treatments and a cure for GNE Myopathy (GNEM/HIBM) through its flagship International Gene Therapy Development Program (IGTDP). It also delivers patient advocacy, education, and outreach programs including a Virtual Learning Library, GNEM Multidisciplinary Clinics, Patient & Carrier Advocacy, Speaker Series, and Genetic Screening services. Its offerings are made available to patients, families, and researchers globally at no charge, supported by a network of Certified Patient Advocates across 50+ countries.

Product overview

The Neuromuscular Disease Foundation (NDF) operates as a nonprofit organization focused on enhancing quality of life for those living with GNE Myopathy (GNEM) through funding critical research and providing advocacy, education, and outreach programs. The organization's offerings include the International Gene Therapy Development Program (IGTDP), its flagship research initiative to develop gene therapy treatments, combined with patient support services such as the Learning Library (featuring podcasts, webinars, and patient stories), GNEM Multidisciplinary Clinics, Patient & Carrier Advocacy programs, and Genetic Screening services. NDF serves patients, caregivers, and researchers across 50+ countries with a team of 43 scientific advisors.

Differentiator

Problem solved

Functional benefit

Products and services

• International Gene Therapy Development Program (IGTDP) NDF's flagship research program that collaborates with world-leading gene therapy and GNEM experts, including Charles River Labs, to develop and begin FDA-approved clinical trials for GNEM using gene therapy.
• Virtual Learning Library A comprehensive digital resource library providing recordings of GNEM Speaker Series events, podcasts, webinars, and patient stories to educate and support those affected by GNE Myopathy.
• GNEM Multidisciplinary Clinic Specialized clinics providing coordinated multidisciplinary care for patients with GNE Myopathy, offering services from various medical specialists.
• Patient & Carrier Advocacy Program Program providing patient resources, support groups, and certified patient advocates to support individuals and families affected by GNE Myopathy through a network of advocates in 50+ countries.
• Genetic Screening Services Genetic testing and screening services to help identify carriers of the GNE gene mutation and support family planning decisions for those affected by GNE Myopathy.
• Speaker Series and Huddles Regular virtual events featuring expert speakers discussing GNEM research, treatments, patient experiences, and disease management topics.

Quantifiable outcome

• 4873 people served annually
• +3 more outcomes

Segments3 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Partnerships

Eleven partnerships are on record, tiered flagship, core and minor.

• Charles River LabsflagshipStrategic or Co-development PartnerCharles River Labs is a pharmaceutical research and drug development leader partnered with NDF for the International Gene Therapy Development Program (IGTDP). This collaboration is central to NDF's flagship initiative to develop a gene therapy for GNE Myopathy and advance it through FDA-approved clinical trials.
• Dr. Stella Mitrani-Rosenbaum (Hadassah Hebrew University Medical Center)coreStrategic or Co-development PartnerDr. Stella Mitrani-Rosenbaum's laboratory continues vital research developing a new mouse model of GNEM essential for testing GNEM therapies.
• Dr. Paul Martin (Abigail Wexner Research Institute at Nationwide Children's Hospital)coreStrategic or Co-development PartnerDr. Paul Martin is developing models of GNEM that support therapeutics development as part of NDF-funded research.
• Drs. Rüdiger Horstkorte and Kaya Bork (Martin-Luther-Universität Halle–Wittenberg)coreStrategic or Co-development PartnerThese researchers can now measure the activity of the GNE protein to determine how well various therapeutics improve GNEM, supported by NDF funding.
• Dr. Kelly Crowe (Xavier University)coreStrategic or Co-development PartnerDr. Kelly Crowe continues her work on lectin staining as a biomarker to measure gene therapy efficacy. NDF sponsored her for the New Investigator Draft Award from Uplifting Athletes.
• Dr. Monkol Lek (Yale School of Medicine)coreStrategic or Co-development PartnerDr. Monkol Lek continues to expand the GNEM patient cell bank, an important resource for researchers. Dr. Lek also leads efforts to develop a gene therapy for GNEM in collaboration with Cure Rare Disease.
• Rich Horgan (Cure Rare Disease)coreStrategic or Co-development PartnerDr. Monkol Lek leads gene therapy development for GNEM in collaboration with Mr. Rich Horgan of Cure Rare Disease.
• Beverly Hills Rotary ClubminorOthersThe Beverly Hills Rotary Club has renewed its support of the Neuromuscular Disease Foundation.
• Whittier TrustminorOthersListed as a sponsor of NDF.
• LeadiantminorOthersListed as a sponsor of NDF.
• Global Advocacy AllianceminorOthersNDF is a founding member of the Global Advocacy Alliance.

Scale indicators5 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Friedreich's Ataxia Research Alliance (FARA): Disease-specific research and patient advocacy nonprofit for Friedreich's ataxia. Comparable in structure: small foundation, patient registry, scientific advisory board, research grants toward rare neuromuscular/neurologic disease therapies.
• ALS Association: Disease-specific nonprofit combining research funding with patient services and advocacy for amyotrophic lateral sclerosis. Comparable in mission structure (rare neuromuscular disease, research grants, multidisciplinary clinics, advocacy network) though at much larger scale.
• A Foundation Building Strength: While lesser known, several small single-disease neuromuscular foundations (e.g., Congenital Muscle Disease Study Group, Myotonic Dystrophy Foundation) operate under a directly analogous model of patient-led gene therapy research funding.
• Charcot-Marie-Tooth Association: Patient advocacy and research nonprofit for Charcot-Marie-Tooth disease, a rare inherited neuromuscular disorder. Comparable patient-support and research-funding model for an ultra-rare neuromuscular condition.
• Parent Project Muscular Dystrophy (PPMD): Disease-specific nonprofit funding Duchenne muscular dystrophy research and advocacy. Highly comparable operating model: parent-led foundation, targeted research grants, gene therapy development focus, and certified patient advocate network.
• Cure SMA: Disease-specific nonprofit driving spinal muscular atrophy research, including successful gene therapy approval pathways (Zolgensma). Closely analogous model of a small foundation that built a gene therapy ecosystem for an ultra-rare neuromuscular disease.

Broad incumbents

• National Organization for Rare Disorders (NORD): Umbrella advocacy and research-support organization for all rare diseases in the U.S. Comparable as a rare-disease nonprofit infrastructure provider and policy advocate, though operating at category level rather than single disease.
• Muscular Dystrophy Association (MDA): Largest U.S. neuromuscular disease nonprofit funding research and providing patient services across dozens of conditions including GNEM/HIBM. Directly comparable as a disease-research and patient-advocacy nonprofit in the neuromuscular space, though much broader in disease scope.
• EveryLife Foundation for Rare Diseases: Rare disease policy and advocacy nonprofit supporting disease-specific foundations. Relevant as an ecosystem-level peer in rare disease advocacy that engages with foundations like NDF on federal policy and FDA engagement.

Emerging players

• Cure Rare Disease: Nonprofit biotech developing gene therapies for ultra-rare diseases, partnered with NDF via Dr. Monkol Lek. Directly relevant as both a peer and active collaborator in NDF's IGTDP gene therapy program for GNEM.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xtwitter.com/curehibm
• YouTubeyoutube.com/channel/UCVWarg-WbNmnpCRdNZ7V0YA
• Facebookfacebook.com/NDF.HIBM
• LinkedInlinkedin.com/company/the-neuromuscular-disease-foundation
• Instagraminstagram.com/ndf.curehibm

Revenue estimate

Valuation estimate

Number of profiles

Profiles9 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about THE NEUROMUSCULAR DISEASE FOUNDATION