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Evaluation of long-term effectiveness of the use of carglumic acid in patients with propionic acidemia (PA) or methylmalonic acidemia (MMA): study protocol for a randomized controlled trial

Crossref DOI link: https://doi.org/10.1186/s12887-019-1571-y

Published Online: 2019-06-13

Published Print: 2019-12

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

Authors

Nashabat, Marwan

Obaid, Abdulrahman

Al Mutairi, Fuad

Saleh, Mohammed

Elamin, Mohammed

Ahmed, Hind

Ababneh, Faroug

Eyaid, Wafaa

Alswaid, Abdulrahman

Alohali, Lina

Faqeih, Eissa

Aljeraisy, Majed

Hussein, Mohamed A.

Alasmari, Ali

Alfadhel, Majid https://orcid.org/0000-0002-9427-7240
Funding

Funding for this research was provided by:

Orphan Europe
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Article History

Received: 12 July 2018

Accepted: 4 June 2019

First Online: 13 June 2019

Ethics approval and consent to participate

: The study was approved by the IRB in the participating centers as follows:IRB at King Abdullah International Medical Research Center (KAIMRC): RC13/116.IRB at King Fahad Medical City (KFMC) (14–165).All the patients were given an approved consent form to participate which was discussed and signed by the patients’ legal guardians before the enrolment.

: The clinical trial and the study medication are funded by Orphan Europe. The scientific design, data management, and data analysis are independent of the funding party.

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