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Editorial
. 2017 Oct 26;377(17):1682-1684.
doi: 10.1056/NEJMe1709253. Epub 2017 Oct 4.

Optimizing Treatment for Cerebral Adrenoleukodystrophy in the Era of Gene Therapy

Marc Engelen  1
Affiliations
Editorial

Optimizing Treatment for Cerebral Adrenoleukodystrophy in the Era of Gene Therapy

Marc Engelen. N Engl J Med. .
No abstract available

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  • Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
    Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA. Eichler F, et al. N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4. N Engl J Med. 2017. PMID: 28976817 Free PMC article. Clinical Trial.

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