EuropeResearch and clinical trials
Stem cell gene therapy in MLD: long-term benefit in children treated before symptoms began
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
Long-term results of atidarsagene autotemcel, a stem cell gene therapy for metachromatic leukodystrophy, have been published. In Milan, 39 treated patients were compared with 49 untreated patients. One group consisted of children with the late infantile type who were treated before symptoms began. All of these children were alive without severe loss of movement at age 6. Among untreated children of the same type, this rate was zero. Three patients died; the researchers did not find these deaths to be related to the treatment.
What does it mean for patients and families?
The benefit of treatment is greatest when it is given before symptoms begin, which shows the importance of early diagnosis and family screening. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes.
Source
Scientific paper: Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy. The New England Journal of Medicine, 2025. DOI: 10.1056/NEJMoa2405727 · PubMed 40267426
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Metachromatic leukodystrophy (MLD)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.