The NHS in England is to have a national service for leukodystrophies following the development of new treatments, it was announced today.
Patients will get virtual clinical reviews and early genetic testing as part of the service, NHS England said. Testing will take place at nationally designated molecular genetics testing with expertise in the diseases.
The launch of the Inherited White Matter Disorders Diagnostic and Management Service follows the first treatment of a child with a gene therapy, Libmeldy, for a metachromatic leukodystrophy, which is normally fatal.
John Stewart, Director for Specialised Commissioning at NHS England said: “This new service is a pioneering model of NHS care, with a combination of virtual and face-to-face care with access to a range of experts.
“This means hundreds of children and adults will see IWMD specialists and get a genetic diagnosis sooner. The new clinical registry also provides opportunities for clinicians to learn more about the condition, identify patients likely to benefit from trials of potential new treatments and will enable patients to share information about how they are feeling”.
Sara Hunt, chief executive of Alex TLC, The Leukodystrophy Charity, said: “The need for improved access to care and treatments for the leukodystrophy community are well known to Alex TLC. This new service and registry will help to alleviate these health inequalities, improve patient outcomes and provide invaluable data with which to promote vital research.
“As patient voice representatives for this project, it has been inspirational to see how valued and influential our community’s priorities have been within development and implementation of the Service and Registry. The impact for patients, and those that care for them, will be significant and we look forward to our continued involvement”.
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