Biobank for Children with Genetic Muscle Diseases

The National Muscle Disease Bio-databank, co-led by Murdoch Children’s Research Institute, Monash University and Alfred Health, will advance research into understanding why children develop genetic muscle diseases. The project forms part of a $2.5 million Medical Research Future Fund grant awarded to the team for research into congenital muscle diseases.

These diseases, spanning dystrophies and myopathies, are characterized by severe muscle weakness, usually from infancy, that can impact swallowing, and breathing and lead to eye problems and learning difficulties. About 30 people a year are diagnosed with a congenital muscular disease in Australia, of which half will have a genetic basis identified.

Murdoch Children’s Dr. Peter Houweling said there was an unmet need for affordable treatments that could be fast-tracked into clinical trials.

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