LGMD Organizations

The Argentinian Muscular Dystrophy Association (ADM) is a non-profit organization that was founded in Argentina in 1983 by a group of people living with neuromuscular diseases (NMD), their family members, and health care providers.  Their mission is focused on improving the quality of life of people living with neuromuscular diseases through qualified care and information, promoting the development of medical capabilities for their care and treatment, and also supporting the research and public policy impact.

In 2020, LGMD-ADM Project was established to offer a national benchmark for people living with LGMD in collaboration with the scientific community.

The Daniel Ferguson Foundation is an Australian non-profit public charity which aims to improve the lives of those living with limb-girdle muscular dystrophy, type 2A/R1 (LGMD2A/R1).

The Foundation bears the name of Daniel Ferguson, a person who has been living with Limb Girdle Muscular Dystrophy Type 2A/R1 for 25+ years, who, with the same drive and commitment he has brought to all aspects of his life, has made it his mission to ensure that others living with LGMD 2A/R1 can live a full and enjoyable life.

The French Muscular Dystrophy Association (AFM-Téléthon Association) is composed of patients and their families who are affected by genetic, rare and progressive diseases that lead to heavy handicaps.

The French LGMD Patient Group was created at the beginning of 2018 and is part of the AFM-Téléthon Association (French Muscular Dystrophy Association). It focuses on all subtypes of limb-girdle muscular dystrophy with the goal to find treatments no matter how long it takes!

“Conquistando Escalones” Association was created for individuals with limb-girdle muscular dystrophy, type IF/D2 (LGMD 1F/D2) and their families. In the past 15 years, great advances have been done in the research on this disease, but the main discover has been done in 2013: the malfunction on gene TNPO3 that causes this disease, makes people affected by LGMD 1F/D2 immune to HIV infection and they could be a possible cure for the AIDS. Since this extremely important discovery, not only for the LGMD 1F/D2 affected but also for millions of peoples, we have got the attention of media and companies. This helped us in making ourselves, and the importance of this research, known and allowed us to organize several events to collect the money we used to directly finance an international research project on LGMD 1F/D2 and AIDS with the objective of healing both of them.

The Family Group of Beta-sarcoglycanopathy (GFB ONLUS) is a group of families with people with beta-sarcoglycanopathy and other limb-girdle muscular dystrophies. THE GFB ONLUS is a non-profit organization and it was created to finance research projects aimed at treating limb-girdle muscular dystrophy type 2E/R4 (LGMD2E/R4).

Formed in February 2013, since 2012, the family of GFB ONLUS has raised more than $ 700.000 for research into LGMD 2E/R4. Among other initiatives, the association is currently funding a gene therapy project about LGMD 2E/R4.

The Italian Association Calpain 3 (AICa3 Onlus) is a non-profit organization with the focus to the fight against limb-girdle muscular dystrophy type 2A/R1 (LGMD 2A/R1) caused by calpain 3 deficiency.

The mission of AICa3 is to support research in order to find the therapeutic treatment that puts a definitive end to limb-girdle muscular dystrophy type 2A/R1 (LGMD 2A/R1) and other calpainopathies. Since this is a very rare disease and consequently almost unknown, we want to stimulate and maintain public interest for individuals that suffer from this disease by promoting and supporting scientific research, by collecting funds for the same, and give help to all those people who are facing this problem through the exchange of information and experience.

The Italian Association of Emery Dreifuss Muscular Dystrophy, also known as AIDMED, is a non-profit social (non-profit) that aims to involve all people affected by this disease and those who want to support us, building a network of solidarity and support in difficult times. Emery Dreifuss Muscular Dystrophy/LGMD1B stems from a genetic mutation causing visible damage to the skeletal muscles and the heart muscle.

Among the objectives of the Association is to support research projects on these genetic mutations. More contributions we can gather, the faster the goals of these research projects will become concrete realities.

UILDM was founded in August 1961 by Federico Milcovich and is the Italian national association of reference for people with dystrophies and other neuromuscular diseases. UILDM LGMD Group doesn’t focus on a specific LGMD sub-type but instead is inclusive to all forms of LGMD. UILDM LGMD Group activities focus on: encouraging the development of a LGMD community; providing scientific and medical information on LGMD; and supporting the exchange of experiences and advice between people that share the same condition.

This association is referred to as ‘Nihon dysferlinopathy kanjya-kai’ in Japanese and PSDJ, ‘Patients’ Association for Dysferlinopathy (MM/LGMD2B/DACM) Japan’ in English.

PADJ was established for the communication between Japanese and international patients with dysferlinopathy (Miyoshi myopathy, Miyoshi muscular dystrophy 1 (MMD1) and limb-girdle muscular dystrophy type 2B/R2 (LGMB 2B/R2), development of early treatment for dysferlinopathy aimed at a complete cure.

Stichting Spierkracht is a non-profit organization based in the Netherlands and is primarily focused on limb-girdle muscular dystrophy type 2D/R3 (LGMD 2D/R3). The organization reaches out to all patients and their families diagnosed with LGMD2D in the Netherlands. The focus is on raising awareness, education, problem-solving and everything you need to know before you enroll in a trial.

Also available is reliable information for the patient’s next of kin with LGMD2D on all adequate subjects in the different stages in the patients’ lives. We also have one qualified patient expert and we work on early diagnosis and patient advisory not only for the Dutch patients but worldwide.

“Conquistando Escalones” Association was created for individuals with limb-girdle muscular dystrophy, type IF/D2 (LGMD 1F/D2) and their families. In the past 15 years, great advances have been done in the research on this disease, but the main discover has been done in 2013: the malfunction on gene TNPO3 that causes this disease, makes people affected by LGMD 1F/D2 immune to HIV infection and they could be a possible cure for the AIDS. Since this extremely important discovery, not only for the LGMD 1F/D2 affected but also for millions of peoples, we have got the attention of media and companies. This helped us in making ourselves, and the importance of this research, known and allowed us to organize several events to collect the money we used to directly finance an international research project on LGMD 1F/D2 and AIDS with the objective of healing both of them.

Proyecto Alpha is a non-profit Spanish association created to help those affected and relatives who suffer from limb-girdle muscular dystrophy due to a deficiency of any of the proteins that make up the Sarcoglycan Type complex: Alpha, Beta, Gamma and Delta)

Beyond Labels & Limitations, a non-profit organization established to raise money dedicated exclusively to limb-girdle muscular dystrophy type 2A/R1 (LGMD 2A/R1) and to educate on the disease course and associated struggles of LGMD 2A/R1, as recounted through my personal experiences.

In 2007 when BL&L was established we wanted the organization to look beyond the current status in both the course of the disease and perceptions of what’s to come; to believe there is a cure out there and we’ll find it; and in hope for what’s to come in the future.

Camron’s Cure Foundation is a non-profit public organization that is focused on uniting people afflicted by this rare disease and in supporting vital research to find a cure and treatment for Limb Girdle Muscular Dystrophy, type 2S/R18 (LGMD 2S/R18).

Coalition to Cure Calpain 3 (C3) has a pinpoint focus: to drive a cure for limb-girdle muscular dystrophy type 2A (LGMD2A/R1), also called Calpainopathy. For the last decade, C3 has been the U.S.-based non-profit leader of funding for LGMD2A/R1 research and is led by Scientific Director Dr. Jennifer Levy who directs the grant program, with a portfolio focused on gene and cell therapy, novel approaches, tool creation, and clinical trial readiness.

CureLGMD2i Foundation is a non-profit public charity and our goal is to support cutting-edge research facilities who are working to establish an effective treatment or cure for limb-girdle muscular dystrophy, type 2I/R9 (LGMD2I/R9).

The CureLGMD2i Foundation was created by the Brazzo Family when their daughter, Samantha, was diagnosed with Limb Girdle Muscular Dystrophy (LGMD Type 2I/R9) at the age of two. At that time, the Brazzos realized that there were no organizations accepting donations specifically for research for LGMD 2I/R9. They decided to create this Nonprofit Organization (formerly known as The Samantha J. Brazzo Foundation) with a mission to spread awareness about LGMD 2I/R9 and to raise funds to support research for this progressive disease.

The Dion Children Foundation for Rare Disease is a non-profit public charity which aims to increase awareness of rare and ultra-rare genetic diseases such as Limb-Girdle Muscular Dystrophy, that affect children with the intention of allocating funds for research and development of potential treatments and cures of these devastating diseases. Our belief at Dion Children Fund is that no child should be left behind. Our children matter, and so do the lives of so many other children affected by rare genetic neuromuscular diseases.

The Jain Foundation is a non-profit private foundation whose mission is to cure muscular dystrophies caused by dysferlin protein deficiency, which includes the clinical presentations limb-girdle muscular dystrophy type 2B/R2 (LGMD 2B/R2) and Miyoshi muscular dystrophy 1 (MMD1). The foundation is privately funded and does not solicit funding from patients or other sources.

The Foundation’s focused strategy includes funding and actively monitoring the progress of scientific research projects in key pathways towards a cure, providing financial and logistical support to promising drug candidates to accelerate them to clinical trials, funding clinical trials and studies, encouraging collaboration among scientists, and educating LGMD 2B/R2 and Miyoshi patients about their disease and helping them with their diagnosis (e.g., funding dysferlin protein and gene mutational analysis).

The Kurt + Peter Foundation, a non-profit public organization, was formed by the family and friends of Kurt and Peter Frewing to raise money and direct it into the hands of researchers who have the best shot at developing a treatment or cure for limb-girdle muscular dystrophy type 2C/R5 (LGMD 2C/R5). Since 2010, the Kurt+Peter Foundation has raised more than $1 million for research into LGMD 2C/R5. Among other initiatives, the foundation is currently funding development of an exon-skipping compound that the foundation hopes will treat the majority of LGMD 2C/R5 mutations.

The LGMD-1D DNAJB6 Foundation is a non-profit public foundation whose mission is to maintain a central location for individuals diagnosed with limb-girdle muscular dystrophy type 1D/D1 (LGMD1D) to meet, consolidate relevant news, and launch fundraising efforts in order to advance research. Genetic and molecular research for these orphan diseases are exploding and techniques that are developed to cure can often be shared with others often afflicted with other crippling genetic disorders that are seemingly unrelated. Our cause is relatively small but our vision includes all people struggling with under-recognized genetic disorder.

The LGMD2D Foundation is a non-profit private foundation whose mission is to expedite the development of a cure or therapy for limb-girdle muscular dystrophy type 2D/R3 (LGMD 2D/R3). In addition to educating patients and physicians, the Foundation maintains a patient registry, funds and monitors research and progress, provides financial support to accelerate clinical trials, and encourages scientific collaboration.

The mission of the LGMD 2D Foundation is to support research and clinical trials to speed the development of treatments and cures for LGMD 2D/R3. Formed in September 2013, the foundation is committed to the development and maintenance of an international patient registry for individuals diagnosed with LGMD 2D/R3 and to supporting relevant scientific research.

The LGMD2L Foundation’s goal is to unite people afflicted by the rare disease limb-girdle muscular dystrophy type 2L/R12 (LGMD 2L/R12). Also, provide an open forum to connect, discuss, and ultimately build a community of LGMD 2L/R12 members. Although our disease is rare and poorly understood, we have established this foundation to build a database of LGMD 2L/R12 patients for future scientific research and clinical trials. The only way to bring about change is to unify those who are driven for a cure!

The LGMD2i Research Fund is a non-profit private foundation whose mission is to expedite the development of a treatment for limb-girdle muscular dystrophy type 2I/R9 (LGMD 2I/R9). Through its grant awards program, the LGMD2I Research Fund promotes basic research, fosters clinical trial readiness and supports the translation of promising scientific discoveries into clinics.

McColl-Lockwood Laboratory for Muscular Dystrophy Research is a non-profit public charity with the mission to develop experimental therapies for the treatment of the muscular dystrophies and to facilitate the translation of experimental therapies to clinical trials in order to improve the quality of life for patients with the disease. Initiated in 2003, the Carolinas Muscular Dystrophy Research Endowment is dedicated to supporting research for select types of muscular dystrophy, particularly Limb-Girdle, which is currently being conducted in the McColl-Lockwood Laboratory for Muscular Dystrophy Research at Carolinas Medical Center, directed by Qi Long Lu, MD, PhD.

Team Titin, Inc. is a nonprofit corporation. Our mission is to support individuals, families, and their healthcare providers, and serve as a catalyst for academic and industry research to develop a better understanding of Titin-related disorders. Changes in the TTN (titin) gene cause a spectrum of muscle and heart disorders that range from rare to common and can have a negative impact on health and quality of life. Titin-related heart conditions are the most common genetic cause of a heart condition called dilated cardiomyopathy. While estimates vary, as many as 1 of 500 adults may have this condition.

The Speak Foundation (TSF) is a non-profit organization that is run entirely by volunteers who live with a form of Muscular Dystrophy or who have a loved one with a disability. We are a charitable, faith-based organization that is dedicated to helping people of any age cope with and overcome the challenges of living with a neuromuscular disease (NMD). We are available to provide support to people with other health conditions as well. We personally understand the challenges that many people with MD face; TSF was created to unite a community, give all people with disabilities a voice, and defend the rights of those in need. A central part of our mission is to reach out to those living with NMDs, to connect their lives through mentorship, provide information and support, and inspire hope. One way that we do this is through our annual summer conference, which provides a context for relationship building and educational sessions on the many issues that affect those with NMDs most deeply. We hope to connect with those who are newly diagnosed so that we can provide you with a community of helpful information and resources that will greatly assist you. The International LGMD Conference is a project of The Speak Foundation.  The LGMD News magazine is published quarterly by TSF.

Breathe with MD, Inc. is a non-profit public organization with the primary goal to educate and raise awareness about breathing muscle weakness in Neuromuscular Disease (NMD). We want those who live with NMD and their loved ones to recognize symptoms and understand intervention options, equipping them to be advocates for their own appropriate respiratory care. We believe knowledge is power and can mean the difference between developing a respiratory crisis which can limit options for care, or proactively managing symptoms and having an enhanced quality of life.

The Clifton R. Lewis Good Life Foundation is a non-profit organization which provides support and assistance to better the lives of people with muscular dystrophy. Through accessibility assistance, special equipment, funding for research and the Dream Creator the foundation will strive to improve the lives of people with MD. “To The Good Life”

Cure VCP Disease, Inc. was formed to drive efforts to cure diseases related to mutations of the Valosin Containing Protein gene. This includes the disease Inclusion Body Myopathy associated with Paget’s disease of bone and Frontotemporal Dementia (IBMPFD). The specific objectives of our organization are: 1) to provide global education and awareness of VCP diseases to doctors, researchers, investors, patients, caregivers and the general public; 2) to develop and maintain a global patient registry of VCP disease patients; 3) to develop and maintain a fundraising vehicle to support awareness and education efforts; 4) to collaborate with other global organizations and entities which are promoting a cure for diseases which have symptoms similar to VCP diseases; 5) to sponsor, host and/or participate in events and activities that promote efforts to cure VCP diseases.

Global Genes is a 501(c)(3) non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally.

Global Genes’ story began with our loved ones. We are the friends, family and supporters of patients close to us who are affected by rare disease. We understand the confusion, the overwhelming experience of the unknown and the feelings of isolation all too well. And we know we aren’t alone. That’s the reason Global Genes was born – to connect, empower and inspire the rare disease community. We fulfill our mission by helping patients find and build communities, gain access to information and resources, connect to researchers, clinicians, industry, government, and other stakeholders, share data and experiences, stand up, stand out, and become effective advocates on their own behalf.

Inclusive is an organization in the United Arab Emirates that supports people living with LGMD and works towards making online and offline environments are accessible for People Of Determination (In 2016 by HH Sheikh Mohammed bin Rashid Al Maktoum introduced the term People Of Determination to describe people who were formally called special needs as outlined in the Federal Law No. 29 of 2006 Concerning the Rights of People with Special Needs in the United Arab Emirates)

Inclusive welcomes everyone to its Online Platform for accessible opportunities in jobs and events. The online platform is for People Of Determination and Supporters Of Inclusion. Inclusive is a for-profit model with a social impact at heart. With a clear focus on revenue generation to enable 15% market of the world, Inclusive is the world’s first tech-enabled startup of its kind.

The Muscular Dystrophy Clinic at Nationwide Children’s is nationally recognized for leading research and clinical care for patients with any of the muscular dystrophy types. Supported in part by the Muscular Dystrophy Association (MDA), we provide the highest level of comprehensive care to patients from across the country.

Our multidisciplinary team is the foundation of our clinic. The combined expertise of specialists in neuromuscular disorders, cardiology, pulmonary medicine, and other disciplines coordinate patient care in a single clinic location. Specialized therapists in the clinic manage activities of daily living and assist patients with techniques to adapt to progressive symptoms. Nationwide Children’s is also an NIH-funded Paul D. Wellstone Muscular Dystrophy Cooperative Research Center.

NMD United was born from the idea that adults with neuromuscular disabilities needed a way to connect, share knowledge, support each other, and empower one another. NMD United is a 501(c)3 non-profit organization governed by a board of adults with neuromuscular disabilities, bringing to the table diverse backgrounds and skill sets, yet all having shared similar experiences that have created a sense of community. Together, they have established NMD United.

TREAT-NMD is a network for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Since its launch in January 2007 the network’s focus has been on the development of tools that industry, clinicians and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide.