Infantile Krabbe disease (0–12 months), progression, and recommended endpoints for clinical trials
Infantile Krabbe disease with onset before 12 months is rapidly progressive. Irreversible brain damage occurs unless timely HSCT is performed. HSCT does not prevent the progression of peripheral nerve disease. This study can be used to monitor patients and evaluate the effects of future therapies.
Authors
- Melissa R. Greco (ORCID: https://orcid.org/0009-0009-4012-7202)
- Ecenur Tuc (ORCID: https://orcid.org/0000-0002-0835-3447)
- Mabel Lopez
- Maria L. Escolar (ORCID: https://orcid.org/0000-0003-1530-6777)
- Maria L. Beltran‐Quintero (ORCID: https://orcid.org/0000-0002-9884-3748)
- Michele D. Poe
Institutions
- University of Pittsburgh (US)
- Biolog (United States) (US)
Publication Details
- Journal
- Annals of Clinical and Translational Neurology
- Published
- 2024-11-05
- DOI
- https://doi.org/10.1002/acn3.52114
- Citations
- 3
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.98