Long-term effectiveness of carglumic acid in patients with propionic acidemia (PA) and methylmalonic acidemia (MMA): a randomized clinical trial

Long-term effectiveness of carglumic acid in patients with propionic acidemia (PA) and methylmalonic acidemia (MMA): a randomized clinical trial

BACKGROUND: Propionic acidemia (PA) and methylmalonic acidemia (MMA) are rare, autosomal recessive inborn errors of metabolism that require life-long medical treatment. The trial aimed to evaluate the effectiveness of the administration of carglumic acid with the standard treatment compared to the s...

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Hoofdauteurs: Alfadhel, Majid, Nashabat, Marwan, Saleh, Mohammed, Elamin, Mohammed, Alfares, Ahmed, Al Othaim, Ali, Umair, Muhammad, Ahmed, Hind, Ababneh, Faroug, Al Mutairi, Fuad, Eyaid, Wafaa, Alswaid, Abdulrahman, Alohali, Lina, Faqeih, Eissa, Almannai, Mohammed, Aljeraisy, Majed, Albdah, Bayan, Hussein, Mohamed A., Rahbeeni, Zuhair, Alasmari, Ali
Formaat: Text
Taal:Engels
Gepubliceerd in: BioMed Central 2021
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Research
Online toegang:https://pmc.ncbi.nlm.nih.gov/articles/PMC8507242/
https://pubmed.ncbi.nlm.nih.gov/34635114
http://dx.doi.org/10.1186/s13023-021-02032-8
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https://pmc.ncbi.nlm.nih.gov/articles/PMC8507242/
https://pubmed.ncbi.nlm.nih.gov/34635114
http://dx.doi.org/10.1186/s13023-021-02032-8

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