Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy

N Engl J Med. 2024 Oct 3;391(13):1256-1258. doi: 10.1056/NEJMc2407165. Epub 2024 Sep 18.

Authors

Lucia Laugwitz¹, Thomas P Mechtler², Nils Janzen³, Petra Oliva², Andrea-Romana Kasper⁴, Charlotte E Teunissen⁵, Friederike Bürger⁶, Joachim Janda⁶, Michaela Döring⁷, Marcus Weitz⁷, Peter Lang⁷, Pascal Martin⁷, Stefanie Beck-Woedl⁷, Charlotte Chanson⁸, Mirko M Essing⁸, Andrew Shenker⁸, Tobias B Haack⁷, Johannes H Schulte⁷, Hendrik Rosewich⁷, Berthold Streubel⁴, David C Kasper², Samuel Groeschel⁷

Affiliations

¹ University Hospital Tübingen, Tübingen, Germany lucia.laugwitz@med.uni-tuebingen.de.
² ARCHIMEDlife Medical Laboratories, Vienna, Austria.
³ Screening Laboratory Hannover, Hannover, Germany.
⁴ Medical University of Vienna, Vienna, Austria.
⁵ Vrije Universiteit Amsterdam, Amsterdam, the Netherlands.
⁶ Heidelberg University, Heidelberg, Germany.
⁷ University Hospital Tübingen, Tübingen, Germany.
⁸ Orchard Therapeutics, London, United Kingdom.

PMID: 39292923
DOI: 10.1056/NEJMc2407165

No abstract available

Publication types

Letter

Grants and funding

421769743/Deutsche Forschungsgemeinschaft