182 related articles for article (PubMed ID: 37606592)
1. Development of precision therapies for rare inborn errors of metabolism: Functional investigations in cell culture models.
Didiasova M; Banning A; Tikkanen R
J Inherit Metab Dis; 2024 May; 47(3):509-516. PubMed ID: 37606592
[TBL] [Abstract][Full Text] [Related]
2. Towards personalized genome-scale modeling of inborn errors of metabolism for systems medicine applications.
Heinken A; El Kouche S; Guéant-Rodriguez RM; Guéant JL
Metabolism; 2024 Jan; 150():155738. PubMed ID: 37981189
[TBL] [Abstract][Full Text] [Related]
3. Computational drug repositioning for rare diseases in the era of precision medicine.
Delavan B; Roberts R; Huang R; Bao W; Tong W; Liu Z
Drug Discov Today; 2018 Feb; 23(2):382-394. PubMed ID: 29055182
[TBL] [Abstract][Full Text] [Related]
4. Developments in evidence creation for treatments of inborn errors of metabolism.
Stockler-Ipsiroglu S; Potter BK; Yuskiv N; Tingley K; Patterson M; van Karnebeek C
J Inherit Metab Dis; 2021 Jan; 44(1):88-98. PubMed ID: 32944978
[TBL] [Abstract][Full Text] [Related]
5. RNA solutions to treat inborn errors of metabolism.
Martinez-Pizarro A; Desviat LR
Mol Genet Metab; 2022 Aug; 136(4):289-295. PubMed ID: 35849888
[TBL] [Abstract][Full Text] [Related]
6. Omics-Based Strategies in Precision Medicine: Toward a Paradigm Shift in Inborn Errors of Metabolism Investigations.
Tebani A; Afonso C; Marret S; Bekri S
Int J Mol Sci; 2016 Sep; 17(9):. PubMed ID: 27649151
[TBL] [Abstract][Full Text] [Related]
7. DDIEM: drug database for inborn errors of metabolism.
Abdelhakim M; McMurray E; Syed AR; Kafkas S; Kamau AA; Schofield PN; Hoehndorf R
Orphanet J Rare Dis; 2020 Jun; 15(1):146. PubMed ID: 32527280
[TBL] [Abstract][Full Text] [Related]
8. An analysis of FDA-approved drugs for metabolic diseases.
Kinch MS; Umlauf S; Plummer M
Drug Discov Today; 2015 Jun; 20(6):648-51. PubMed ID: 25680688
[TBL] [Abstract][Full Text] [Related]
9. Inborn errors of metabolism at the turn of the millennium.
Barić I; Fumić K; Hoffmann GF
Croat Med J; 2001 Aug; 42(4):379-83. PubMed ID: 11471189
[TBL] [Abstract][Full Text] [Related]
10. Research challenges in central nervous system manifestations of inborn errors of metabolism.
Dickson PI; Pariser AR; Groft SC; Ishihara RW; McNeil DE; Tagle D; Griebel DJ; Kaler SG; Mink JW; Shapiro EG; Bjoraker KJ; Krivitzky L; Provenzale JM; Gropman A; Orchard P; Raymond G; Cohen BH; Steiner RD; Goldkind SF; Nelson RM; Kakkis E; Patterson MC
Mol Genet Metab; 2011 Mar; 102(3):326-38. PubMed ID: 21176882
[TBL] [Abstract][Full Text] [Related]
11. Neurocognitive clinical outcome assessments for inborn errors of metabolism and other rare conditions.
Shapiro E; Bernstein J; Adams HR; Barbier AJ; Buracchio T; Como P; Delaney KA; Eichler F; Goldsmith JC; Hogan M; Kovacs S; Mink JW; Odenkirchen J; Parisi MA; Skrinar A; Waisbren SE; Mulberg AE
Mol Genet Metab; 2016 Jun; 118(2):65-9. PubMed ID: 27132782
[TBL] [Abstract][Full Text] [Related]
12. Complex patterns of inheritance, including synergistic heterozygosity, in inborn errors of metabolism: Implications for precision medicine driven diagnosis and treatment.
Vockley J; Dobrowolski SF; Arnold GL; Guerrero RB; Derks TGJ; Weinstein DA
Mol Genet Metab; 2019; 128(1-2):1-9. PubMed ID: 31358473
[TBL] [Abstract][Full Text] [Related]
13. Genotype-based databases for variants causing rare diseases.
Lanthaler B; Wieser S; Deutschmann A; Schossig A; Fauth C; Zschocke J; Witsch-Baumgartner M
Gene; 2014 Oct; 550(1):136-40. PubMed ID: 25111118
[TBL] [Abstract][Full Text] [Related]
14. Contemporary scope of inborn errors of metabolism involving epilepsy or seizures.
Tumienė B; Peterlin B; Maver A; Utkus A
Metab Brain Dis; 2018 Dec; 33(6):1781-1786. PubMed ID: 30006695
[TBL] [Abstract][Full Text] [Related]
15. Precision medicine for rare diseases: The times they are A-Changin'.
Amaral MD
Curr Opin Pharmacol; 2022 Apr; 63():102201. PubMed ID: 35255452
[TBL] [Abstract][Full Text] [Related]
16. The individual (single patient) IND for inborn errors of metabolism.
Dickson PI; Tolar J
Mol Genet Metab; 2015; 116(1-2):1-3. PubMed ID: 25456747
[TBL] [Abstract][Full Text] [Related]
17. Integrative methods for analyzing big data in precision medicine.
Gligorijević V; Malod-Dognin N; Pržulj N
Proteomics; 2016 Mar; 16(5):741-58. PubMed ID: 26677817
[TBL] [Abstract][Full Text] [Related]
18. Clinical experience with orphan drugs for rare metabolic diseases.
Caso-González A; Núñez-Rodríguez J; Nebot-Villacampa MJ; González-Pérez Y; Marín-Gorricho R; Leralta-González C; Obaldia-Alaña C
An Pediatr (Engl Ed); 2022 Jan; 96(1):8-16. PubMed ID: 34992005
[TBL] [Abstract][Full Text] [Related]
19. Development of medicines for rare diseases and inborn errors of metabolism: Toward novel public-private partnerships.
Rosenberg N; Stolwijk NN; van den Berg S; Heus JJ; van der Wel V; van Gelder T; Bosch AM; de Visser SJ; Hollak CEM
J Inherit Metab Dis; 2023 Sep; 46(5):806-816. PubMed ID: 36938792
[TBL] [Abstract][Full Text] [Related]
20. The treatable intellectual disability APP www.treatable-id.org: a digital tool to enhance diagnosis & care for rare diseases.
van Karnebeek CD; Houben RF; Lafek M; Giannasi W; Stockler S
Orphanet J Rare Dis; 2012 Jul; 7():47. PubMed ID: 22824307
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]