MCDS-Therapy aims repurpose a drug to treat the pain and bone malformations in a rare skeletal condition

MCDS-Therapy is EU-funded clinical trial testing whether carbamazepine can be repurposed to treat the rare genetic bone disorder, metaphyseal chondrodysplasia type Schmid (MCDS). BM Manifesto is proud to be one of the project’s 11 partners from across the globe, with responsibility for communication and dissemination about the project. As part of this role we plan to engage as many people as possible in this exciting research.

What is MCDS?

MCDS, or metaphyseal chondrodysplasia type Schmid, is an ultra-rare inherited disorder. It leads to short stature with disproportionately short arms and legs (called short-limbed dwarfism), as well as curvature, or bowing, of the leg bones (known as genu varum). The ends of long bones and ribs can also flare outwards, and there may be hip deformities causing the thigh bone to be angled towards the centre of the body (known as coxa vara). These characteristics can lead to an impaired gait when walking that can make movement difficult, as well as pain in the legs and joints throughout life.

Find out more about MCDS-Therapy

The MCDS-Therapy consortium plans to develop a low cost treatment that will alleviate the pain and bone malformations commonly experienced by patients, and will work closely with patients to do so. The project has been funded by the European Union’s Horizon 2024 Research and Innovation programme for 5 years.

You can learn more about the inception of this project by watching Prof Mike Briggs’ talk at BM Manifesto’s Drug Repurposing for Rare Diseases 2018 conference, or by visiting the MCDS-Therapy website.