Karen presented at the ‘Leukodystrophies: Novel perspectives on treatments and disease monitoring’ course in Paris. The event was held by Professor Fanny Mouchel and attended by many leukodystrophy specialists – including paediatric and adult neurologists, neuroradiologists, paediatricians, geneticists and neuroscientists.
Karen presented about how Alex TLC supports families and newborn screening for ALD and MLD – we are grateful to Nala and Teddi’s mum for allowing us to share their journey as part of the presentation. Karen will also discuss how research is vital for families to access treatment and using the recent case where an individual with a rare leukodystrophy was able to access treatment in the UK following the publication of the findings of a research study.
