What LGMD2L Foundation does

The LGMD2L Foundation is a 501(c)(3) nonprofit patient advocacy organization founded in 2018 and headquartered in Princeton, New Jersey, that unites a global community of individuals affected by LGMD2L (also called LGMDR12), a rare genetic disorder causing progressive limb-girdle muscle weakness and affecting up to 1 in 60,000 people worldwide. The foundation's mission expanded in 2024 from awareness and community building to actively funding research toward treatments, and it now operates a portfolio of research initiatives including a flagship gene replacement therapy program for ANO5-related disease developed with Cure Rare Disease under a $7.65 million multi-year commitment, a foundational mouse model at Virginia Commonwealth University, an international muscle regeneration study at Copenhagen University Hospital jointly funded with MDA, FSHD Society, and Parent Project Muscular Dystrophy, and organoid drug repurposing and autologous CRISPR cell therapy investigations at Université Paris-Est Créteil and Charité University Medicine Berlin.

The foundation's core 'products' are research funding flowing to academic and biotech partners, a patient registry built with Citizen Health that collects real-world data and medical records for research and pharmaceutical use, and community support resources delivered through a website, social media channels (Facebook, Instagram, LinkedIn), email, and patient storytelling. It does not sell products or services; revenue is generated entirely through tax-deductible donations processed via PayPal Giving Fund, individual fundraising campaigns, and grants, with documented multi-year commitments and campaign totals exceeding $8 million across recent programs. The foundation is governed by a board led by Founder and President Ralph Yaniz, with VP Research Hal Tily, PhD, and a Scientific Advisory Committee comprising researchers from Emory University, UCLA, Washington University, Satellos, and the Muscular Dystrophy Association.

Name

LGMD2L Foundation

Legal name

LGMD2L Foundation

Website

https://www.lgmd2l-foundation.org

Company type

Private

Founded year

2018

Operating status

Operating

Headcount range

1–10 employees

Short description

The LGMD2L Foundation is a 501(c)(3) nonprofit founded in 2018 that funds research, maintains a patient registry, and supports individuals affected by LGMD2L, a rare ANO5-related muscular dystrophy affecting up to 1 in 60,000 people worldwide.

Ownership category

akta.pro rank

Product category

Rare Disease Patient Advocacy Foundation

NAICS

Voluntary Health Organizations (813212)

SIC

Services-Commercial Physical & Biological Research (8731), Services-Health Services (8000)

akta.pro primary industry

Health & Medical Research Grantmaking Foundations (BPAGAKAL)

akta.pro secondary industry

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

Keywords

• Rare disease research
• Patient registry services
• Muscular dystrophy advocacy
• Gene therapy funding
• Nonprofit health foundation

Headquarters

HQ city

Princeton

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, Technology or R&D, Others

Revenue model

1. Individual Donations: The foundation raises funds from individual donors, families, and patients affected by LGMD2L. Recent fundraising campaigns include a $25,000+ campaign for mouse model development and ongoing campaigns for gene therapy research.
2. Partnership Funding: Major partnership funding including $7.65 million committed from the LGMD2L Foundation to Cure Rare Disease for multi-year gene therapy development.
3. Tax-Deductible Donations: All donations are processed through PayPal Giving Fund and are tax-deductible in the USA. Donations support critical research and advocacy efforts.

Go-to-market motion1 record

Distribution channels4 records

Marketing channels8 records

Core offering

The LGMD2L Foundation is a patient-led 501(c)(3) nonprofit that funds foundational and translational research into LGMD2L (ANO5-related muscular dystrophy), operates a digital patient registry in partnership with Citizen Health to collect real-world data, and supports individuals and families affected by the disease. It directs multi-year research grants to academic and biotech partners working on gene therapy, mouse models, muscle regeneration, and drug repurposing for LGMD2L.

Product overview

The LGMD2L Foundation operates as a nonprofit patient advocacy organization offering a patient registry (in partnership with Citizen Health) that collects real-world data to support research and clinical trials, along with multiple research funding initiatives including a major gene replacement therapy program developed in partnership with Cure Rare Disease, mouse model development at Virginia Commonwealth University, muscle regeneration research with international coalition partners, and drug repurposing screening projects.

Differentiator

Problem solved

Functional benefit

Products and services

• LGMD2L Registry A patient registry developed in partnership with Citizen Health that collects real-world data from patients living with LGMD2L, securely gathers medical records from healthcare providers, and enables data sharing with researchers and pharmaceutical companies studying disease progression and treatment responses.
• Gene Replacement Therapy Program for LGMD2L A multi-year development program funded by the foundation in partnership with Cure Rare Disease to develop a novel gene replacement therapy for Anoctamin 5 (ANO5)-related disease (LGMD2L/R12), covering therapeutic design, preclinical studies, manufacturing scale-up, and a first-in-human clinical trial.
• LGMD2L Mouse Model Development Project A research project funded by the foundation ($31,233 raised) at Virginia Commonwealth University to develop a new mouse model for ANO5-related disease, enabling preclinical testing of treatments and supporting future approval of treatments for human use.
• Muscle Regeneration Research Initiative A collaborative research initiative with MDA, FSHD Society, and Parent Project Muscular Dystrophy to create a molecular map of how healthy human skeletal muscle regenerates, with a $213,446 grant awarded to Dr. Abigail Mackey at Copenhagen University Hospital analyzing muscle repair at single-nucleus resolution.
• Drug Repurposing Screening A foundation-funded research project at Université Paris-Est Créteil examining how existing compounds affect LGMD2L patient muscles by deriving small muscle-like organoids from patient cells for compound screening and drug repurposing.

Quantifiable outcome

• Successfully raised $31,233 for mouse model development enabling treatment testing capabilities
• +3 more outcomes

Named customers1 record

Segments3 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Partnerships

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

• Cure Rare Disease (CRD)flagshipStrategic or Co-development Partner · 2 March 2026Multi-year partnership backed by $7.65 million in funding from the LGMD2L Foundation to develop a novel gene replacement therapy for Anoctamin 5 (ANO5)-related disease. The program supports therapeutic design, preclinical studies, manufacturing scale-up, and a first-in-human clinical trial. CRD's pipeline already includes gene therapies for related conditions LGMD2I/R9 and LGMD2G/R7, enabling accelerated development for LGMD2L/R12.
• Muscular Dystrophy Association (MDA)coreStrategic or Co-development Partner · 27 January 2026Joint research initiative to create the most detailed molecular map of how healthy human skeletal muscle regenerates and how the process is altered in neuromuscular diseases including LGMD2L, Duchenne, and FSHD. MDA's 2024 Muscle Regeneration Summit identified this as a priority. A $213,446 grant was awarded to Dr. Abigail Mackey at Copenhagen University Hospital.
• FSHD SocietycoreStrategic or Co-development Partner · 27 January 2026International coalition partner in the muscle regeneration research initiative alongside MDA, Parent Project Muscular Dystrophy, and the LGMD2L Foundation. Collaboratively funding research at Copenhagen University Hospital to analyze muscle repair at single-nucleus resolution.
• Parent Project Muscular Dystrophy (PPMD)coreStrategic or Co-development Partner · 27 January 2026Joint funding partner in the muscle regeneration research collaboration with MDA and FSHD Society. Grant of $213,446 awarded to Copenhagen University Hospital for research expected to serve as foundational resource for regenerative therapies across multiple muscle disorders.
• Citizen HealthcoreTechnology or IntegrationTechnology partnership for the LGMD2L Registry platform. Citizen Health provides the digital infrastructure to collect real-world patient data, securely contact healthcare providers to gather medical records, and enable data sharing with researchers and pharmaceutical companies.
• Virginia Commonwealth UniversitycoreStrategic or Co-development PartnerResearch partnership for developing a new mouse model for ANO5-related disease. The project, funded by the foundation's $25,000+ fundraiser (total $31,233), will enable testing of existing and new treatments and support approval for human clinical trials.
• Copenhagen University HospitalcoreStrategic or Co-development PartnerResearch institution leading the international muscle regeneration study. Dr. Abigail Mackey directs the project analyzing muscle repair at single-nucleus resolution in healthy and disease-affected tissue, funded by a $213,446 grant from the coalition including the LGMD2L Foundation.
• Université Paris-Est CréteilminorStrategic or Co-development PartnerBoard member Axel Kruger funded a project examining how existing compounds affect LGMD2L patient muscles. The work derives small muscle-like organoids from patient cells to screen a large number of compounds for repurposing potential.
• Charite, University Medicine BerlinminorStrategic or Co-development PartnerResearch partnership funded by board member Axel Kruger for autologous cell therapy research. The project explores methods to extract muscle stem cells from patients, correct the mutation using CRISPR, and reintroduce cells to build healthy muscle.

Scale indicators5 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• Cure Rare Disease: Nonprofit biotech developing gene replacement therapies for ultra-rare muscular dystrophies. CRD is both the foundation's flagship partner and a direct peer — same patient-led nonprofit model, same gene therapy modality, and overlapping LGMD disease scope (CRD also works on LGMD2I/R9 and LGMD2G/R7).
• Parent Project Muscular Dystrophy (PPMD): Disease-specific nonprofit focused on Duchenne muscular dystrophy that funds research, maintains a patient registry, and runs multi-foundation coalitions. Closely mirrors the LGMD2L Foundation's model — single-disease focus, registry, academic grantmaking, and joint coalition funding.
• FSHD Society: Disease-specific research and advocacy foundation for facioscapulohumeral muscular dystrophy. Operates with the same patient-led grantmaking model, funds academic research, and is a current LGMD2L Foundation coalition partner in the muscle regeneration initiative.
• Jain Foundation: Nonprofit focused specifically on LGMD2B (dysferlinopathy) that funds research, supports patient registries, and drives therapeutic development for a single LGMD subtype. Direct functional peer — same disease-specific grantmaking, registry, and therapy-development approach.
• Coalition to Cure Calpain 3 (C3): Patient-led nonprofit funding research toward treatments for LGMD2A/R1 (calpainopathy). Operates with a virtually identical model — small team, focused on a single LGMD subtype, funds academic and biotech research, maintains patient engagement.
• Friedreich's Ataxia Research Alliance (FARA): Disease-specific nonprofit for Friedreich's ataxia that funds research, supports a patient registry (FA Global Patient Registry), and coordinates multi-stakeholder therapeutic development. Same playbook applied to a different rare neuromuscular disease.
• Charley's Fund: Family-founded nonprofit funding Duchenne muscular dystrophy gene therapy development. Mirrors the LGMD2L Foundation's focus on accelerating gene therapy in a rare muscular dystrophy through dedicated grantmaking to research partners.

Broad incumbents

• Muscular Dystrophy Association (MDA): The largest US voluntary health organization covering neuromuscular diseases including LGMDs. MDA is both a coalition partner and a broad peer — same disease-advocacy mission and grantmaking activity but operates across many conditions rather than focusing on a single subtype.

Others

• Satellos Bioscience: Public biotech developing muscle regeneration therapeutics for Duchenne and LGMDs. Listed as a member of the foundation's Scientific Advisory Committee and represents the industry/biotech counterpart to the foundation's grantmaking model.
• Citizens Health Foundation / Citizen Health: Digital health platform powering the LGMD2L Registry. Provides patient-registry infrastructure used by multiple rare disease communities, making it an enabling peer rather than a competing foundation.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xtwitter.com/lgmd2l
• Facebookfacebook.com/LGMD2L-Foundation
• LinkedInlinkedin.com/company/lgmd2l
• Instagraminstagram.com/lgmd2l.foundation

Revenue estimate

Valuation estimate

Number of profiles

Profiles5 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about LGMD2L Foundation