The road to therapy

MDC1A / Lama2 CMD is a very rare, congenital muscle disease affecting patients worldwide. Extensive research is needed to develop a cure. On this website we inform you about the patient organisations that fight for research and the various scientific studies that are ongoing. Together we fight for a treatment and a healthier future. Join the cause, stay informed, or donate.

What is MDC1A?

What exactly is MDC1A?

Babies and children suffering from MDC1A / Lama2 CMD have difficulty walking and breathing due to muscle weakness. Among these symptoms, patients generally suffer from a lower quality of life. The disease is caused by rare mutations in LAMA2, a gene responsible for the protein that holds muscles together.

Although symptoms can be reduced with physiotherapy, currently there is no cure due to its rare nature and limited research opportunities.

More about MDC1a

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2019 conference

Watch the 2019 MDC1A conference


Stay up to date

  • Dr. Avencia Sanchez-Mejías’ ex-vivo therapeutic approach to MDC1A

    Dr. Avencia Sanchez-Mejías has been investigating how haematopoietic stem cells from MDC1A patients’ bone marrow, can be genetically corrected to potentially improve quality of life. Dr. Avencia Sanchez-Mejías and her research team at the University of Pompeu Fabra in Barcelona have carried out a number of investigations focused on an ex-vivo therapeutic approach to MDC1A.…

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  • Get to know the work of respiratory therapist Ruud van der Wel

    As a respiratory therapist and an innovator, Ruud van der Wel has been able to develop amazing tools that can transform respiratory therapy into interactive games for children with MDC1A / Lama2 CMD.  Ruud van der Wel is a Dutch respiratory therapist at Rijndam Rehabilitation Institute that has been working with children with disabilities for…

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Do you have a question for one of the experts or researchers with whom we are in contact? About an investigation, a treatment method or other information that you are looking for? Submit your question and we will get you a personal answer as soon as possible. We will also publish the answer on this website.