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

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Title: Long-term effectiveness of carglumic acid in patients with propionic acidemia (PA) and methylmalonic acidemia (MMA): a randomized clinical trial.
Authors: Alfadhel, Majid1 (AUTHOR) dralfadhelm@gmail.com, Nashabat, Marwan1 (AUTHOR), Saleh, Mohammed2 (AUTHOR), Elamin, Mohammed2 (AUTHOR), Alfares, Ahmed3 (AUTHOR), Al Othaim, Ali4 (AUTHOR), Umair, Muhammad5 (AUTHOR), Ahmed, Hind1 (AUTHOR), Ababneh, Faroug1 (AUTHOR), Al Mutairi, Fuad1 (AUTHOR), Eyaid, Wafaa1 (AUTHOR), Alswaid, Abdulrahman1 (AUTHOR), Alohali, Lina1 (AUTHOR), Faqeih, Eissa2 (AUTHOR), Almannai, Mohammed2 (AUTHOR), Aljeraisy, Majed6 (AUTHOR), Albdah, Bayan7 (AUTHOR), Hussein, Mohamed A.7 (AUTHOR), Rahbeeni, Zuhair8 (AUTHOR), Alasmari, Ali2 (AUTHOR)
Source: Orphanet Journal of Rare Diseases. 10/11/2021, Vol. 16 Issue 1, p1-10. 10p.
Database: Academic Search Ultimate
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ISSN:17501172
DOI:10.1186/s13023-021-02032-8