From scientific discovery to treatments for rare diseases – the view from the National Center for Advancing Translational Sciences – Office of Rare Diseases Research-Reference-Cited by-同舟云学术

From scientific discovery to treatments for rare diseases – the view from the National Center for Advancing Translational Sciences – Office of Rare Diseases Research

Published:2018-11-06 Issue:1 Volume:13 Page:
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet J Rare Dis

Author:

Kaufmann Petra,Pariser Anne R.,Austin Christopher

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics (clinical),General Medicine

Link

http://link.springer.com/content/pdf/10.1186/s13023-018-0936-x.pdf

Reference27 articles.

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3. Mendell JR, Al-Zaidy S, Shelll R, Arnold WD, Rodino-Klapac LR, Prior TW, et al. Single-dose gene replacement therapy for spinal muscular atrophy. N Engl J Med. 2017;377:1713–22.

4. Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani G, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med. 2009;360(5):447–58.

5. Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, et al. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase ½ tiral. Lancet. 2016;388:476–87.

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