186 related articles for article (PubMed ID: 31119742)
1. Pathophysiology of propionic and methylmalonic acidemias. Part 2: Treatment strategies.
Haijes HA; van Hasselt PM; Jans JJM; Verhoeven-Duif NM
J Inherit Metab Dis; 2019 Sep; 42(5):745-761. PubMed ID: 31119742
[TBL] [Abstract][Full Text] [Related]
2. Pathophysiology of propionic and methylmalonic acidemias. Part 1: Complications.
Haijes HA; Jans JJM; Tas SY; Verhoeven-Duif NM; van Hasselt PM
J Inherit Metab Dis; 2019 Sep; 42(5):730-744. PubMed ID: 31119747
[TBL] [Abstract][Full Text] [Related]
3. Retrospective evaluation of the Dutch pre-newborn screening cohort for propionic acidemia and isolated methylmalonic acidemia: What to aim, expect, and evaluate from newborn screening?
Haijes HA; Molema F; Langeveld M; Janssen MC; Bosch AM; van Spronsen F; Mulder MF; Verhoeven-Duif NM; Jans JJM; van der Ploeg AT; Wagenmakers MA; Rubio-Gozalbo ME; Brouwers MCGJ; de Vries MC; Langendonk JG; Williams M; van Hasselt PM
J Inherit Metab Dis; 2020 May; 43(3):424-437. PubMed ID: 31828787
[TBL] [Abstract][Full Text] [Related]
4. 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.
Nashabat M; Obaid A; Al Mutairi F; Saleh M; Elamin M; Ahmed H; Ababneh F; Eyaid W; Alswaid A; Alohali L; Faqeih E; Aljeraisy M; Hussein MA; Alasmari A; Alfadhel M
BMC Pediatr; 2019 Jun; 19(1):195. PubMed ID: 31196016
[TBL] [Abstract][Full Text] [Related]
5. Methylmalonic acidemia/propionic acidemia - the biochemical presentation and comparing the outcome between liver transplantation versus non-liver transplantation groups.
Chu TH; Chien YH; Lin HY; Liao HC; Ho HJ; Lai CJ; Chiang CC; Lin NC; Yang CF; Hwu WL; Lee NC; Lin SP; Liu CS; Hu RH; Ho MC; Niu DM
Orphanet J Rare Dis; 2019 Apr; 14(1):73. PubMed ID: 30940196
[TBL] [Abstract][Full Text] [Related]
6. Liver transplantation in propionic and methylmalonic acidemia: A single center study with literature review.
Pillai NR; Stroup BM; Poliner A; Rossetti L; Rawls B; Shayota BJ; Soler-Alfonso C; Tunuguntala HP; Goss J; Craigen W; Scaglia F; Sutton VR; Himes RW; Burrage LC
Mol Genet Metab; 2019 Dec; 128(4):431-443. PubMed ID: 31757659
[TBL] [Abstract][Full Text] [Related]
7. Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia.
Chandler RJ; Venditti CP
J Inherit Metab Dis; 2024 Jan; 47(1):63-79. PubMed ID: 37530705
[TBL] [Abstract][Full Text] [Related]
8. Biomarkers for drug development in propionic and methylmalonic acidemias.
Longo N; Sass JO; Jurecka A; Vockley J
J Inherit Metab Dis; 2022 Mar; 45(2):132-143. PubMed ID: 35038174
[TBL] [Abstract][Full Text] [Related]
9. New insights into the pathophysiology of methylmalonic acidemia.
Head PE; Meier JL; Venditti CP
J Inherit Metab Dis; 2023 May; 46(3):436-449. PubMed ID: 37078237
[TBL] [Abstract][Full Text] [Related]
10. Proposed guidelines for the diagnosis and management of methylmalonic and propionic acidemia.
Baumgartner MR; Hörster F; Dionisi-Vici C; Haliloglu G; Karall D; Chapman KA; Huemer M; Hochuli M; Assoun M; Ballhausen D; Burlina A; Fowler B; Grünert SC; Grünewald S; Honzik T; Merinero B; Pérez-Cerdá C; Scholl-Bürgi S; Skovby F; Wijburg F; MacDonald A; Martinelli D; Sass JO; Valayannopoulos V; Chakrapani A
Orphanet J Rare Dis; 2014 Sep; 9():130. PubMed ID: 25205257
[TBL] [Abstract][Full Text] [Related]
11. Long-term liver disease in methylmalonic and propionic acidemias.
Imbard A; Garcia Segarra N; Tardieu M; Broué P; Bouchereau J; Pichard S; de Baulny HO; Slama A; Mussini C; Touati G; Danjoux M; Gaignard P; Vogel H; Labarthe F; Schiff M; Benoist JF
Mol Genet Metab; 2018 Apr; 123(4):433-440. PubMed ID: 29433791
[TBL] [Abstract][Full Text] [Related]
12. An Early Cost-Utility Model of mRNA-Based Therapies for the Treatment of Methylmalonic and Propionic Acidemia in the United Kingdom.
Bretos-Azcona PE; Wallace M; Jootun M; Jin G; Agirrezabal I; Szende A
Clin Drug Investig; 2024 Jun; 44(6):399-412. PubMed ID: 38796677
[TBL] [Abstract][Full Text] [Related]
13. Cost-effectiveness of liver transplantation in methylmalonic and propionic acidemias.
Li M; Dick A; Montenovo M; Horslen S; Hansen R
Liver Transpl; 2015 Sep; 21(9):1208-18. PubMed ID: 25990417
[TBL] [Abstract][Full Text] [Related]
14. Acute Illness Protocol for Organic Acidemias: Methylmalonic Acidemia and Propionic Acidemia.
Aldubayan SH; Rodan LH; Berry GT; Levy HL
Pediatr Emerg Care; 2017 Feb; 33(2):142-146. PubMed ID: 28141776
[TBL] [Abstract][Full Text] [Related]
15. Understanding acute metabolic decompensation in propionic and methylmalonic acidemias: a deep metabolic phenotyping approach.
Haijes HA; Jans JJM; van der Ham M; van Hasselt PM; Verhoeven-Duif NM
Orphanet J Rare Dis; 2020 Mar; 15(1):68. PubMed ID: 32143654
[TBL] [Abstract][Full Text] [Related]
16. Long-term effectiveness of carglumic acid in patients with propionic acidemia (PA) and methylmalonic acidemia (MMA): a randomized clinical trial.
Alfadhel M; Nashabat M; Saleh M; Elamin M; Alfares A; Al Othaim A; Umair M; Ahmed H; Ababneh F; Al Mutairi F; Eyaid W; Alswaid A; Alohali L; Faqeih E; Almannai M; Aljeraisy M; Albdah B; Hussein MA; Rahbeeni Z; Alasmari A
Orphanet J Rare Dis; 2021 Oct; 16(1):422. PubMed ID: 34635114
[TBL] [Abstract][Full Text] [Related]
17. Liver and/or kidney transplantation in amino and organic acid-related inborn errors of metabolism: An overview on European data.
Molema F; Martinelli D; Hörster F; Kölker S; Tangeraas T; de Koning B; Dionisi-Vici C; Williams M;
J Inherit Metab Dis; 2021 May; 44(3):593-605. PubMed ID: 32996606
[TBL] [Abstract][Full Text] [Related]
18. Guidelines for the diagnosis and management of methylmalonic acidaemia and propionic acidaemia: First revision.
Forny P; Hörster F; Ballhausen D; Chakrapani A; Chapman KA; Dionisi-Vici C; Dixon M; Grünert SC; Grunewald S; Haliloglu G; Hochuli M; Honzik T; Karall D; Martinelli D; Molema F; Sass JO; Scholl-Bürgi S; Tal G; Williams M; Huemer M; Baumgartner MR
J Inherit Metab Dis; 2021 May; 44(3):566-592. PubMed ID: 33595124
[TBL] [Abstract][Full Text] [Related]
19. Liver Transplantation for Propionic Acidemia and Methylmalonic Acidemia: Perioperative Management and Clinical Outcomes.
Critelli K; McKiernan P; Vockley J; Mazariegos G; Squires RH; Soltys K; Squires JE
Liver Transpl; 2018 Sep; 24(9):1260-1270. PubMed ID: 30080956
[TBL] [Abstract][Full Text] [Related]
20. Challenges and strategies for clinical trials in propionic and methylmalonic acidemias.
Vockley J; Burton B; Jurecka A; Ganju J; Leiro B; Zori R; Longo N
Mol Genet Metab; 2023 Jul; 139(3):107612. PubMed ID: 37245378
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]