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Posted: 2025-04-24 16:49:56 UTC

This article contains some claims that remain unverified. While much of the content may be accurate, exercise care when relying on this information.
This article contains some claims that remain unverified. While much of the content may be accurate, exercise care when relying on this information.
Status
Last Updated
2025-04-24 16:50:13 UTC
Verified By
Rollup News
A study comparing clinical outcomes of 39 patients treated with atidarsagene autotemcel, a gene therapy for metachromatic leukodystrophy (MLD), to untreated patients demonstrated a significant and positive long-term treatment effect.
Atidarsagene autotemcel shows a strong treatment effect for MLD.
The study provides long-term outcome data for gene therapy in MLD patients.