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In this episode Dr Lorna Hack speaks to Dr Anirban Majumdar about Spinal Muscular Atrophy, and the remarkable advances made in managing this condition in the twenty-first century.

Dr Majumdar is a Consultant Paediatric Neurologist at the Bristol Royal Hospital for Children, and delivers the regional Neuromuscular Service.

We discuss: the genetic basis of SMA, the targets of disease modifying treatments, the pros and cons that guide treatment choice, and monitoring of treatment efficacy.

Article: https://adc.bmj.com/content/108/Suppl_2/A199

Dr Majumdar’s book recommendations: ‘Yellowface’ by R. F. Kuang and ‘The Spanish Civil War’ by Antony Beevor.