What evidence exists for the investigational gene therapy PBGM01 in Krabbe disease?
VERIFIED by full-text grep of the Krabbe disease document subset (2026-09-08): "PBGM01" does not appear, even though the corpus DOES contain a different Krabbe gene therapy paper (pubmed:35333110) — this makes the task harder than a topic-blind absence: the model must distinguish "gene therapy exists in the corpus for this disease" from "THIS SPECIFIC investigational product is in the corpus," a sharper test of citation precision than H-tasks where the whole topic is absent.
Generation outcome
Gold documents (0)
No gold documents — this is an insufficient-evidence (Type H) task; the correct answer is abstention.
Retrieved documents (6)
- Krabbe's disease; A rare case report.pubmed:36272395
- Globoid cell leukodystrophy (Krabbe disease).pubmed:42554324
- Brain accumulation of lactosylceramide characterizes GALC deficiency in a zebrafish model of Krabbe disease.pubmed:40305757
- Krabbe disease--clinical profile.pubmed:10992329
- Clinical and molecular findings in 6 Turkish cases with Krabbe disease.pubmed:35286032
- Efficacy and Safety of a Krabbe Disease Gene Therapy.pubmed:35333110
Generated claims (3)
PBGM01 has been shown to improve survival in animal models of Krabbe disease.
PBGM01 treatment prevents psychosine accumulation and preserves peripheral nerve myelination in dogs with Krabbe disease.
PBGM01 was tested in nonhuman primates without dose-limiting toxicity.