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The following term was not found in PubMed: Jestratijevic
Page 1
Mucopolysaccharidosis IVA: Diagnosis, Treatment, and Management.
Sawamoto K, Álvarez González JV, Piechnik M, Otero FJ, Couce ML, Suzuki Y, Tomatsu S. Sawamoto K, et al. Int J Mol Sci. 2020 Feb 23;21(4):1517. doi: 10.3390/ijms21041517. Int J Mol Sci. 2020. PMID: 32102177 Free PMC article. Review.
Mucopolysaccharidosis type IVA (MPS IVA, or Morquio syndrome type A) is an inherited metabolic lysosomal disease caused by the deficiency of the N-acetylglucosamine-6-sulfate sulfatase enzyme. ...GAG accumulation in these lesions leads to unique skeletal dysplasia i …
Mucopolysaccharidosis type IVA (MPS IVA, or Morquio syndrome type A) is an inherited metabolic lysosomal disease caused by the …
Faricimab for Treatment-Resistant Diabetic Macular Edema.
Rush RB, Rush SW. Rush RB, et al. Clin Ophthalmol. 2022 Aug 24;16:2797-2801. doi: 10.2147/OPTH.S381503. eCollection 2022. Clin Ophthalmol. 2022. PMID: 36042912 Free PMC article.
PURPOSE: To assess the short-term outcomes in treatment-resistant diabetic macular edema (DME) patients changed from intravitreal aflibercept (IVA) to intravitreal faricimab (IVF). METHODS: A retrospective review was undertaken on DME subjects receiving IVA therapy …
PURPOSE: To assess the short-term outcomes in treatment-resistant diabetic macular edema (DME) patients changed from intravitreal aflibercep …
Intravitreal Faricimab for Aflibercept-Resistant Neovascular Age-Related Macular Degeneration.
Rush RB, Rush SW. Rush RB, et al. Clin Ophthalmol. 2022 Dec 9;16:4041-4046. doi: 10.2147/OPTH.S395279. eCollection 2022. Clin Ophthalmol. 2022. PMID: 36532820 Free PMC article.
PURPOSE: To evaluate the short-term effects of intravitreal faricimab (IVF) in treatment-resistant neovascular age-related macular degeneration (nAMD) subjects previously treated with intravitreal aflibercept (IVA). METHODS: A retrospective review was conducted on nAMD pat …
PURPOSE: To evaluate the short-term effects of intravitreal faricimab (IVF) in treatment-resistant neovascular age-related macular degenerat …
Mucopolysaccharidosis Type IVA.
Regier DS, Oetgen M, Tanpaiboon P. Regier DS, et al. 2013 Jul 11 [updated 2021 Jun 17]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. 2013 Jul 11 [updated 2021 Jun 17]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. PMID: 23844448 Free Books & Documents. Review.
CLINICAL CHARACTERISTICS: The phenotypic spectrum of mucopolysaccharidosis IVA (MPS IVA) is a continuum that ranges from a severe and rapidly progressive early-onset form to a slowly progressive later-onset form. ...Compression of the spinal cord is a common complic …
CLINICAL CHARACTERISTICS: The phenotypic spectrum of mucopolysaccharidosis IVA (MPS IVA) is a continuum that ranges from a sev …
Mucopolysaccharidosis IVA: Current Disease Models and Drawbacks.
Leal AF, Alméciga-Díaz CJ, Tomatsu S. Leal AF, et al. Int J Mol Sci. 2023 Nov 9;24(22):16148. doi: 10.3390/ijms242216148. Int J Mol Sci. 2023. PMID: 38003337 Free PMC article. Review.
Mucopolysaccharidosis IVA (MPS IVA) is a rare disorder caused by mutations in the N-acetylgalactosamine-6-sulfate-sulfatase (GALNS) encoding gene. ...This manuscript reviews the current in vitro and in vivo MPS IVA models and their drawbacks....
Mucopolysaccharidosis IVA (MPS IVA) is a rare disorder caused by mutations in the N-acetylgalactosamine-6-sulfate-sulfatase (G …
Bone Growth Induction in Mucopolysaccharidosis IVA Mouse.
Rintz E, Herreño-Pachón AM, Celik B, Nidhi F, Khan S, Benincore-Flórez E, Tomatsu S. Rintz E, et al. Int J Mol Sci. 2023 Jun 8;24(12):9890. doi: 10.3390/ijms24129890. Int J Mol Sci. 2023. PMID: 37373036 Free PMC article.
Mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome) is caused by a deficiency of the N-acetylgalactosamine-6-sulfate-sulfatase (GALNS) enzyme, leading to the accumulation of glycosaminoglycans (GAG), keratan sulfate (KS) and chondroitin-6-sulfate (C6S), mainly i …
Mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome) is caused by a deficiency of the N-acetylgalactosamine-6-sulfate-sulfa …
Elosulfase alfa.
Haddley K. Haddley K. Drugs Today (Barc). 2014 Jul;50(7):475-83. doi: 10.1358/dot.2014.50.7.2177904. Drugs Today (Barc). 2014. PMID: 25101330 Review.
Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome, is an inherited, lysosomal storage disorder caused by genetic mutations in N-acetylgalactosamine-6-sulfatase (GALNS) enzyme gene. ...BioMarin Pharmaceutical developed elosulfase alfa, a reco …
Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome, is an inherited, lysosomal storage disorder caused …
Diagnosing mucopolysaccharidosis IVA.
Wood TC, Harvey K, Beck M, Burin MG, Chien YH, Church HJ, D'Almeida V, van Diggelen OP, Fietz M, Giugliani R, Harmatz P, Hawley SM, Hwu WL, Ketteridge D, Lukacs Z, Miller N, Pasquali M, Schenone A, Thompson JN, Tylee K, Yu C, Hendriksz CJ. Wood TC, et al. J Inherit Metab Dis. 2013 Mar;36(2):293-307. doi: 10.1007/s10545-013-9587-1. Epub 2013 Feb 1. J Inherit Metab Dis. 2013. PMID: 23371450 Free PMC article.
Mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome) is an autosomal recessive lysosomal storage disorder resulting from a deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS) activity. ...Urinary glycosaminoglycan analysis is particularly problematic …
Mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome) is an autosomal recessive lysosomal storage disorder resulting from a …
Elexacaftor/Tezacaftor/Ivacaftor Treatment and Depression-related Events.
Ramsey B, Correll CU, DeMaso DR, McKone E, Tullis E, Taylor-Cousar JL, Chu C, Volkova N, Ahluwalia N, Waltz D, Tian S, Mall MA. Ramsey B, et al. Am J Respir Crit Care Med. 2024 Feb 1;209(3):299-306. doi: 10.1164/rccm.202308-1525OC. Am J Respir Crit Care Med. 2024. PMID: 37890129 Free PMC article. Review.
After U.S. approval in 2019, reports emerged of depression-related adverse events in pwCF treated with ELX/TEZ/IVA. Objectives: To review available evidence on depression-related events in pwCF treated with ELX/TEZ/IVA in the context of background epidemiology in pw …
After U.S. approval in 2019, reports emerged of depression-related adverse events in pwCF treated with ELX/TEZ/IVA. Objectives: To re …
Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor-Ivacaftor.
Tümmler B. Tümmler B. Front Pharmacol. 2023 Mar 21;14:1158207. doi: 10.3389/fphar.2023.1158207. eCollection 2023. Front Pharmacol. 2023. PMID: 37025483 Free PMC article. Review.
Triple combination therapy with the CFTR modulators elexacaftor (ELX), tezacaftor (TEZ) and ivacaftor (IVA) has been qualified as a game changer in cystic fibrosis (CF). We provide an overview of the body of literature on ELX/TEZ/IVA published between November 2019 …
Triple combination therapy with the CFTR modulators elexacaftor (ELX), tezacaftor (TEZ) and ivacaftor (IVA) has been qualified as a g …
9,691 results