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.
Marwan NashabatAbdulrahman ObaidFuad Al MutairiMohammed SalehMohammed ElaminHind AhmedFaroug AbabnehWafaa EyaidAbdulrahman AlswaidLina AlohaliEissa FaqeihMajed AljeraisyMohamed A HusseinAli AlasmariMajid AlfadhelPublished in: BMC pediatrics (2019)
King Abdullah International Medical Research Center ( KAIMRC ): (RC13/116) 09/1/2014. Saudi Food and Drug Authority (SFDA) (33066) 08/14/2014. ClinicalTrials.gov (identifier: NCT02426775) 04/22/2015.