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Prcoeedings of the Royal Society of Medicine a Form of Progressive

Prcoeedings of the Royal Society of Medicine a Form of Progressive

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[Music] hello my name is Sarah hunt and I'm here today to talk to you about the patient experience of the withdrawal of gene therapy for adrenal lucad distrophy and the reasons behind why the aod patient community so desperately need gen therapy in the UK so Adrina lucad distrophy or aod is the most prevalent of a group of inherited neurod degenerative disorders called the lucad distrophy which affect the white matter of the brain and spinal cord it's an exlink Disorder so most severely affects males and there are several phenotypes affecting children and adults the most severe of which is the cerebral form which is thought to affect at least 45% of males with Al and can develop in childhood or adulthood males with Al are monitored for signs of disease progression in the brain via MRI scan at regular intervals from the age of two with an incidence of one in 15,000 we would expect to see about 40 babies a year born in England based on latest on first statistics in the UK Al is only trea

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