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• Infantile Krabbe disease (0-12 months), progression, and recommended endpoints for clinical trials.

  Greco MR, Lopez MA, Beltran-Quintero ML, Tuc Bengur E, Poe MD, Escolar ML. Greco MR, et al. Ann Clin Transl Neurol. 2024 Dec;11(12):3064-3080. doi: 10.1002/acn3.52114. Epub 2024 Nov 5. Ann Clin Transl Neurol. 2024. PMID: 39499628 Free PMC article.
• A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life.

  Bascou N, DeRenzo A, Poe MD, Escolar ML. Bascou N, et al. Orphanet J Rare Dis. 2018 Aug 9;13(1):126. doi: 10.1186/s13023-018-0872-9. Orphanet J Rare Dis. 2018. PMID: 30089515 Free PMC article.
• Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months.

  Beltran-Quintero ML, Bascou NA, Poe MD, Wenger DA, Saavedra-Matiz CA, Nichols MJ, Escolar ML. Beltran-Quintero ML, et al. Orphanet J Rare Dis. 2019 Feb 18;14(1):46. doi: 10.1186/s13023-019-1018-4. Orphanet J Rare Dis. 2019. PMID: 30777126 Free PMC article.
• A lipid nanoparticle-based oligodendrocyte-specific mRNA therapy.

  Sawamura M, Tachikawa K, Hikawa R, Akiyama H, Kaji S, Yasuda K, Leu AI, Hong H, Mukthavaram R, Chivukula P, Yamakado H, Hirabayashi Y, Takahashi R, Matsuzawa SI. Sawamura M, et al. Mol Ther Nucleic Acids. 2024 Nov 5;35(4):102380. doi: 10.1016/j.omtn.2024.102380. eCollection 2024 Dec 10. Mol Ther Nucleic Acids. 2024. PMID: 39640012 Free PMC article.
• Clinical, electrophysiological, and biochemical markers of peripheral and central nervous system disease in canine globoid cell leukodystrophy (Krabbe's disease).

  Bradbury AM, Bagel JH, Jiang X, Swain GP, Prociuk ML, Fitzgerald CA, O'Donnell PA, Braund KG, Ory DS, Vite CH. Bradbury AM, et al. J Neurosci Res. 2016 Nov;94(11):1007-17. doi: 10.1002/jnr.23838. J Neurosci Res. 2016. PMID: 27638585 Free PMC article.