Major investment to fund Fragile X research

The Medicines Discovery Institute has received a grant of almost £2.5m from the Medical Research Council to improve therapies for the most common inherited cause of learning disabilities.

Professor Simon Ward, Director of the Medicines Discovery Institute at Cardiff University, said “Fragile X syndrome is the most common single genetic mutation that causes autism, affecting around 1 in 5000 males and 1 in 8000 females. The genetic mutation can cause a wide range of difficulties with learning, as well as social, language, attentional, emotional, and behavioural problems. It is our aim to find better treatments that allow patients with fragile X to manage their condition in the way that is best for them.”

Currently, people with fragile X syndrome have to take many medications to manage specific symptoms, such as seizures, attention deficit, hyperarousal, aggression, anxiety, depression and sleep disturbances.

Cardiff University says the research aims to discover a new drug that will inhibit the activity of a protein, LIMK1, which is known to be involved in the process of development of the condition.

Simon Ward said “By targeting this protein, we aim to develop a novel medication that will make a difference to the lives of people living with the condition, as well as their families. We have been working closely with the Fragile X Society and the FRAXA charity to improve treatment options.”

Becky Hardiman, CEO of the Fragile X Society, said “A drug discovery project such as the one proposed may provide valuable insights into potential future drugs which may benefit our community. We are delighted that this innovative project is taking place in the UK and look forward to working with the team to communicate developments to the Fragile X community in the UK.”

Michael Tranfaglia MD, Chief Scientific Officer of FRAXA Research Foundation, said “We are thrilled to see such significant funding for this exciting new approach to fragile X therapeutics. FRAXA has been supportive of this research, as it offers the possibility of truly disease-modifying treatment and it is both innovative and sophisticated in its methods. We will be following the progress of this important work very closely.”

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