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Understanding LGMD

Limb-girdle muscular dystrophy (LGMD) is a group of diseases that cause weakness and wasting of the muscles in the arms and legs.

How can gene therapy help?

Myonexus aims to deliver the first-ever corrective treatments for LGMD through the promise of gene therapy technology. Gene therapy is ideally suited to address rare genetic diseases based on its precision delivery of genetic material.

Community Resources

There are a number of organizations with valuable resources and information for patients with LGMD:

GFB ONLUS is group of families with people with beta-sarcoglycanopathy and other LGMDs. The group was created to finance research projects aimed at treating the disease.

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The Jain Foundation funds basic and translational research focused on understanding and curing dysferlinopathy. The foundation has evolved into a leading player in the dysferlin field.

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Kurt + Peter Foundation was formed by Kurt and Peter’s family and friends to raise money and direct it into the hands of researchers who have the best shot at developing a treatment or cure for LGMD2C.

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LDMD2D Foundation

The LGMD2D Foundation is a registered 501(c)3 non-profit foundation whose mission is to expedite the development of a cure or therapy for Limb-girdle Muscular Dystrophy 2D (LGMD2D).

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Additional educational resources about gene therapy are available through the American Society of Cell and Gene Therapy. Learn more here.

Our Affiliations

We work closely with community foundations to support families and seek insights that help to inform and guide our ambition to transform care.