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Long-term effectiveness of carglumic acid in patients with propionic acidemia (PA) and methylmalonic acidemia (MMA): a randomized clinical trial.
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.
Afiliação
  • Alfadhel M; Genetics and Precision Medicine department (GPM), King Abdullah Specialized Children's Hospital (KASCH), King Abdullah International Medical Research Center, King Saud Bin Abdulaziz University for Health Sciences (KSAUHS), King Abdulaziz Medical City, Ministry of National Guard Health Affairs (MNG-H
  • Nashabat M; Genetics and Precision Medicine department (GPM), King Abdullah Specialized Children's Hospital (KASCH), King Abdullah International Medical Research Center, King Saud Bin Abdulaziz University for Health Sciences (KSAUHS), King Abdulaziz Medical City, Ministry of National Guard Health Affairs (MNG-H
  • Saleh M; Medical Genetics Section, King Fahad Medical City, Children's Hospital, Riyadh, Kingdom of Saudi Arabia.
  • Elamin M; Medical Genetics Section, King Fahad Medical City, Children's Hospital, Riyadh, Kingdom of Saudi Arabia.
  • Alfares A; Department of Pediatrics, College of Medicine, Qassim University, Buraidah, Kingdom of Saudi Arabia.
  • Al Othaim A; Department of Pathology, King Abdullah International Medical Research Centre, King Saud bin Abdulaziz University for Health Science, King Abdulaziz Medical City, Ministry of National Guard-Health Affairs (NGHA), Riyadh, Kingdom of Saudi Arabia.
  • Umair M; Medical Genomics Research Department, King Abdullah International Medical Research Center (KAIMRC), King Saud bin Abdulaziz University for Health Sciences, King AbdulAziz Medical City, Ministry of National Guard Health Affairs, Riyadh, Kingdom of Saudi Arabia.
  • Ahmed H; Genetics and Precision Medicine department (GPM), King Abdullah Specialized Children's Hospital (KASCH), King Abdullah International Medical Research Center, King Saud Bin Abdulaziz University for Health Sciences (KSAUHS), King Abdulaziz Medical City, Ministry of National Guard Health Affairs (MNG-H
  • Ababneh F; Genetics and Precision Medicine department (GPM), King Abdullah Specialized Children's Hospital (KASCH), King Abdullah International Medical Research Center, King Saud Bin Abdulaziz University for Health Sciences (KSAUHS), King Abdulaziz Medical City, Ministry of National Guard Health Affairs (MNG-H
  • Al Mutairi F; Genetics and Precision Medicine department (GPM), King Abdullah Specialized Children's Hospital (KASCH), King Abdullah International Medical Research Center, King Saud Bin Abdulaziz University for Health Sciences (KSAUHS), King Abdulaziz Medical City, Ministry of National Guard Health Affairs (MNG-H
  • Eyaid W; Genetics and Precision Medicine department (GPM), King Abdullah Specialized Children's Hospital (KASCH), King Abdullah International Medical Research Center, King Saud Bin Abdulaziz University for Health Sciences (KSAUHS), King Abdulaziz Medical City, Ministry of National Guard Health Affairs (MNG-H
  • Alswaid A; Genetics and Precision Medicine department (GPM), King Abdullah Specialized Children's Hospital (KASCH), King Abdullah International Medical Research Center, King Saud Bin Abdulaziz University for Health Sciences (KSAUHS), King Abdulaziz Medical City, Ministry of National Guard Health Affairs (MNG-H
  • Alohali L; Genetics and Precision Medicine department (GPM), King Abdullah Specialized Children's Hospital (KASCH), King Abdullah International Medical Research Center, King Saud Bin Abdulaziz University for Health Sciences (KSAUHS), King Abdulaziz Medical City, Ministry of National Guard Health Affairs (MNG-H
  • Faqeih E; Medical Genetics Section, King Fahad Medical City, Children's Hospital, Riyadh, Kingdom of Saudi Arabia.
  • Almannai M; Medical Genetics Section, King Fahad Medical City, Children's Hospital, Riyadh, Kingdom of Saudi Arabia.
  • Aljeraisy M; King Abdullah International Medical Research Centre, College of Pharmacy, King Saud bin Abdulaziz University for Health Science, King Abdulaziz Medical City, Ministry of National Guard-Health Affairs, Riyadh, Kingdom of Saudi Arabia.
  • Albdah B; Department Biostatistics and Bioinformatics, King Abdullah International Medical Research Centre, King Saud bin Abdulaziz University for Health Science, Ministry of National Guard-Health Affairs, Riyadh, Kingdom of Saudi Arabia.
  • Hussein MA; Department Biostatistics and Bioinformatics, King Abdullah International Medical Research Centre, King Saud bin Abdulaziz University for Health Science, Ministry of National Guard-Health Affairs, Riyadh, Kingdom of Saudi Arabia.
  • Rahbeeni Z; Department of Medical Genetics, King Faisal Specialist Hospital and Research Center, Riyadh, Kingdom of Saudi Arabia.
  • Alasmari A; Medical Genetics Section, King Fahad Medical City, Children's Hospital, Riyadh, Kingdom of Saudi Arabia.
Orphanet J Rare Dis ; 16(1): 422, 2021 10 11.
Article em En | MEDLINE | ID: mdl-34635114
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Acidemia Propiônica / Erros Inatos do Metabolismo dos Aminoácidos Tipo de estudo: Clinical_trials / Observational_studies / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Acidemia Propiônica / Erros Inatos do Metabolismo dos Aminoácidos Tipo de estudo: Clinical_trials / Observational_studies / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article