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Effect of carglumic acid with or without ammonia scavengers on hyperammonaemia in acute decompensation episodes of organic acidurias

Crossref DOI link: https://doi.org/10.1186/s13023-018-0840-4

Published Online: 2018-06-20

Published Print: 2018-12

Update policy: https://doi.org/10.1007/springer_crossmark_policy

Authors

Chakrapani, Anupam

Valayannopoulos, Vassili

Segarra, Nuria García

Del Toro, Mireia

Donati, Maria Alice

García-Cazorla, Angeles

González, María Julieta

Plisson, Celine

Giordano, Vincenzo
Funding

Funding for this research was provided by:

Orphan Europe
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Article History

Received: 13 July 2017

Accepted: 11 June 2018

First Online: 20 June 2018

Ethics approval and consent to participate

: The study protocols and amendments were approved by the local independent ethics committees (IECs) and/or institutional review boards. Written informed consent/assent was obtained before data were collected. Cases in which it was not possible to obtain consent (due to death, loss to follow-up) were handled on a case-by-case basis with the relevant IEC. The studies were conducted in accordance with the principles of the Declaration of Helsinki.

: Not applicable.

: VV received Principal Investigator fees from Orphan Europe for conducting this study while at Necker-Enfants Malades Hospital. VV is currently an employee of Sanofi Genzyme Corporation, Cambridge, MA. CP, JLM and VG are employees of Orphan Europe.

: Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.