What are the treatment options for MLD?
We are committed to supporting continuing research to treat symptomatic and non-symptomatic patients, to providing resources and newborn screening so that patients are diagnosed as soon as possible to benefit from treatment, and to providing resources to doctors and loved ones caring for those with MLD.
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Gene Therapy Resources
Gene therapy to treat MLD is being advanced by Orchard Therapeutics, a Kyowa Kirin company. They are now treating individuals commercially in Europe and in the United States. Orchard's gene therapy has mostly been used to treat pre-symptomatic late-infantile MLD and select early juvenile patients.
Most of the patients who have undergone the treatment were diagnosed before showing any symptoms of MLD. At this time, the majority of children treated have been the younger siblings of children diagnosed with late-infantile onset MLD.
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Gene Therapy Resource Hub
MLD Voices | Photo Gallery from MLD on the Hill, May 5th - 8th, 2025
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