← All tasks
Task H-009 · Type H — Insufficient evidence (should abstain)
What clinical trial evidence exists for atidarsagene autotemcel (Lenmeldy) gene therapy in metachromatic leukodystrophy?
Difficulty
medium
Rarity stratum
medium
Evidence type
insufficient_evidence
MONDO ID
MONDO:0018868
Curator notes
VERIFIED by full-text grep of the metachromatic leukodystrophy document subset (2026-09-08): neither "atidarsagene" nor "lenmeldy" appear.
Generation outcome
Abstained
No
Abstention correct
No
Gold in retrieved
n/a (no gold docs)
Generation cost
$0.000386
Claims
3
Supported
2
Contradicted
1
Unverifiable
0
Gold documents (0)
No gold documents — this is an insufficient-evidence (Type H) task; the correct answer is abstention.
Retrieved documents (6)
- Treatment of leukodystrophies: Advances and challenges.pubmed:40279833
- Encapsulated cells as an enzyme replacement therapy for metachromatic leukodystrophy.pubmed:41083008
- Metachromatic leukodystrophy--an update.pubmed:20571983
- A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy.pubmed:34882002
- Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States.pubmed:38613540
- Cross-species efficacy of AAV-mediated ARSA replacement for metachromatic leukodystrophy.pubmed:40536808
Generated claims (3)
supportedconfidence 0.90
Ex vivo gene therapy using atidarsagene autotemcel is recommended for presymptomatic patients with early-onset MLD.
pubmed:38613540
supportedconfidence 0.80
Animal studies have shown that AAV.GMU01-ARSA-mediated gene replacement results in therapeutic ARSA expression in the brain of nonhuman primates.
pubmed:40536808
contradictedconfidence 0.80
Atidarsagene autotemcel is considered the gold standard for pre-symptomatic patients with late-infantile and early juvenile MLD.
pubmed:41083008