Posted By A. Domenighetti


Cerebral palsy (CP) is caused by a primary brain injury that occurs around birth and it is one of the major cause of body movement limitation in children. CP has an enduring impact on the development of the muscles after birth. As the child grows, muscles get shorter, thinner, weaker and less flexible, which can make it considerably harder to move and live an active, healthy and independent life. Our research showed that a drug that is currently approved to treat certain forms of blood cancers in adults and children, can be potentially adapted (“repurposed”) to help grow muscles in children with CP. In a controlled dose-escalation study, we will study the preliminary safety, biological properties and efficacy of this drug, called 5-azacytidine (AZA). Our hope and expectations are that one day AZA will become a new approved pharmacological treatment to support muscle growth and improve body movement and quality of life in children with CP.


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