The road to therapy

MDC1A is a very rare, congenital muscle disease affecting children worldwide. Extensive research is needed to develop a cure. We fight for a treatment and inform you about research and therapy. Join the cause, attend our next conference, or donate.

What is MDC1A?

What exactly is MDC1A?

Babies and children suffering from MDC1A 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


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  • Meet GYM: the Generate your Muscle group

    Under supervision of prof. dr. Bert Smeets, the Generate your Muscle (GYM) group was formed and is in cooperation with Stichting Voor Sara organizing the conference in November. GYM is a group of ambitious students from Maastricht University participating in the FHML Honours Programme. This is an interdisciplinary programme at the University of Maastricht providing…

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