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Page 1
Urine N-Acetylaspartate Distinguishes Phenotypes in Canavan Disease.
Nagy A, Eichler F, Bley A, Bredow J, Fay A, Townsend EL, Leiro B, Shaywitz A, Laforet G, Crippen-Harmon D, Williams R. Nagy A, et al. Among authors: eichler f. Hum Gene Ther. 2024 Jan;36(1-2):45-56. doi: 10.1089/hum.2024.168. Epub 2024 Dec 4. Hum Gene Ther. 2024. PMID: 39628365
An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs.
Vasireddy V, Maguire CA, Anderson DW, Ng C, Gong Y, Eichler F, Fourcade S, Guilera C, Onieva A, Sanchez A, Leal-Julià M, Verdés S, Dijkstra IME, Kemp S, Park H, Lutz T, Clark SW, Bosch A, Pujol A, Kozarsky K. Vasireddy V, et al. Among authors: eichler f. Mol Ther Methods Clin Dev. 2024 Oct 9;32(4):101354. doi: 10.1016/j.omtm.2024.101354. eCollection 2024 Dec 12. Mol Ther Methods Clin Dev. 2024. PMID: 39524975 Free PMC article.
Characterizing visual processing deficits in cerebral adrenoleukodystrophy.
Corre CS, Bambery M, Bennett CR, Nagy A, Manley CE, Winter E, Peregoy C, Kelly D, Andonian H, Maciel S, Becker C, Merabet LB, Eichler FS. Corre CS, et al. Among authors: eichler fs. Brain Dev. 2024 Nov;46(10):344-350. doi: 10.1016/j.braindev.2024.09.008. Epub 2024 Oct 12. Brain Dev. 2024. PMID: 39396893
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy.
Eichler F, Duncan CN, Musolino PL, Lund TC, Gupta AO, De Oliveira S, Thrasher AJ, Aubourg P, Kühl JS, Loes DJ, Amartino H, Smith N, Folloni Fernandes J, Sevin C, Sankar R, Hussain SA, Gissen P, Dalle JH, Platzbecker U, Downey GF, McNeil E, Demopoulos L, Dietz AC, Thakar HL, Orchard PJ, Williams DA. Eichler F, et al. N Engl J Med. 2024 Oct 10;391(14):1302-1312. doi: 10.1056/NEJMoa2400442. N Engl J Med. 2024. PMID: 39383459 Clinical Trial.
Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy.
Duncan CN, Bledsoe JR, Grzywacz B, Beckman A, Bonner M, Eichler FS, Kühl JS, Harris MH, Slauson S, Colvin RA, Prasad VK, Downey GF, Pierciey FJ, Kinney MA, Foos M, Lodaya A, Floro N, Parsons G, Dietz AC, Gupta AO, Orchard PJ, Thakar HL, Williams DA. Duncan CN, et al. Among authors: eichler fs. N Engl J Med. 2024 Oct 10;391(14):1287-1301. doi: 10.1056/NEJMoa2405541. N Engl J Med. 2024. PMID: 39383458 Clinical Trial.
Practical Approach to Longitudinal Neurologic Care of Adults With X-Linked Adrenoleukodystrophy and Adrenomyeloneuropathy.
Kornbluh AB, Baldwin A, Fatemi A, Vanderver A, Adang LA, Van Haren K, Sampson J, Eichler FS, Sadjadi R, Engelen M, Orthmann-Murphy JL. Kornbluh AB, et al. Among authors: eichler fs. Neurol Genet. 2024 Oct 3;10(5):e200192. doi: 10.1212/NXG.0000000000200192. eCollection 2024 Oct. Neurol Genet. 2024. PMID: 39372123 Free PMC article. Review.
173 results