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Quoted phrase not found in phrase index: "Mucopolysaccharidosis, MPS-III-D"
Page 1
Laronidase.
[No authors listed] [No authors listed] BioDrugs. 2002;16(4):316-8. doi: 10.2165/00063030-200216040-00009. BioDrugs. 2002. PMID: 12196045 Review.
BioMarin and Genzyme General have completed a pivotal, Phase III trial in the centres in the USA, Canada and Europe, including patients with Hurler-Scheie and Scheie syndromes. ...In the open-label extension study, patients from both the treatment and placebo arms o …
BioMarin and Genzyme General have completed a pivotal, Phase III trial in the centres in the USA, Canada and Europe, including …
Novel therapies for mucopolysaccharidosis type III.
Seker Yilmaz B, Davison J, Jones SA, Baruteau J. Seker Yilmaz B, et al. J Inherit Metab Dis. 2021 Jan;44(1):129-147. doi: 10.1002/jimd.12316. Epub 2020 Sep 28. J Inherit Metab Dis. 2021. PMID: 32944950 Free PMC article. Review.
Mucopolysaccharidosis type III (MPS III) or Sanfilippo disease is an orphan inherited lysosomal storage disease and one of the most common MPS subtypes. ...Here, we review the numerous approaches of curative therapy developed for MPS III from historica …
Mucopolysaccharidosis type III (MPS III) or Sanfilippo disease is an orphan inherited lysosomal storage disease and one …
A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II.
Okuyama T, Eto Y, Sakai N, Nakamura K, Yamamoto T, Yamaoka M, Ikeda T, So S, Tanizawa K, Sonoda H, Sato Y. Okuyama T, et al. Mol Ther. 2021 Feb 3;29(2):671-679. doi: 10.1016/j.ymthe.2020.09.039. Epub 2020 Sep 30. Mol Ther. 2021. PMID: 33038326 Free PMC article. Clinical Trial.
In order to establish its efficacy and safety, a multicenter, single-arm, open-label phase 2/3 clinical trial was conducted in 28 Japanese patients with mucopolysaccharidosis II (MPS-II, Hunter syndrome) by intravenous administrations of 2.0 mg/kg of pabinafu …
In order to establish its efficacy and safety, a multicenter, single-arm, open-label phase 2/3 clinical trial was conducted in …
Treatment of brain disease in the mucopolysaccharidoses.
Scarpa M, Orchard PJ, Schulz A, Dickson PI, Haskins ME, Escolar ML, Giugliani R. Scarpa M, et al. Mol Genet Metab. 2017 Dec;122S:25-34. doi: 10.1016/j.ymgme.2017.10.007. Epub 2017 Oct 16. Mol Genet Metab. 2017. PMID: 29153844 Free article. Review.
The mucopolysaccharidosis (MPS) disorders are a group of lysosomal storage diseases caused by lysosomal enzyme deficits that lead to glycosaminoglycan accumulation, affecting various tissues throughout the body based on the specific enzyme deficiency. ...There are establis …
The mucopolysaccharidosis (MPS) disorders are a group of lysosomal storage diseases caused by lysosomal enzyme deficits that lead to …
Brain Targeting in MPS-IIIA.
Sorrentino NC, Fraldi A. Sorrentino NC, et al. Pediatr Endocrinol Rev. 2016 Jun;13 Suppl 1:630-8. Pediatr Endocrinol Rev. 2016. PMID: 27491210 Review.
These approaches are highly invasive and poorly suited for clinical use. Minimally invasive approaches are based on systemic injections into the blood stream of therapeutics capable of crossing the blood-brain barrier (BBB). ...Future clinical applications of this a …
These approaches are highly invasive and poorly suited for clinical use. Minimally invasive approaches are based on systemic injectio …
Update of treatment for mucopolysaccharidosis type III (sanfilippo syndrome).
Kong W, Yao Y, Zhang J, Lu C, Ding Y, Meng Y. Kong W, et al. Eur J Pharmacol. 2020 Dec 5;888:173562. doi: 10.1016/j.ejphar.2020.173562. Epub 2020 Sep 16. Eur J Pharmacol. 2020. PMID: 32949598 Review.
Mucopolysaccharidosis III (Sanfilippo syndrome, MPS III) is caused by lysosomal enzyme deficiency, which is a rare autosomal recessive hereditary disease. For now, there is no approved treatment for MPS III despite lots of efforts providing new vision …
Mucopolysaccharidosis III (Sanfilippo syndrome, MPS III) is caused by lysosomal enzyme deficiency, which is a rare auto …
Practical management of behavioral problems in mucopolysaccharidoses disorders.
Escolar ML, Jones SA, Shapiro EG, Horovitz DDG, Lampe C, Amartino H. Escolar ML, et al. Mol Genet Metab. 2017 Dec;122S:35-40. doi: 10.1016/j.ymgme.2017.09.010. Epub 2017 Sep 27. Mol Genet Metab. 2017. PMID: 29170079 Free article. Review.
The mucopolysaccharidosis (MPS) disorders are caused by deficiencies of specific lysosomal enzymes, resulting in progressive glycosaminoglycan (GAG) accumulation in cells and tissues throughout the body. ...Behavioral problems (like hyperactivity, attention difficulties, a …
The mucopolysaccharidosis (MPS) disorders are caused by deficiencies of specific lysosomal enzymes, resulting in progressive glycosam …
Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations.
Parini R, Deodato F. Parini R, et al. Int J Mol Sci. 2020 Apr 23;21(8):2975. doi: 10.3390/ijms21082975. Int J Mol Sci. 2020. PMID: 32340185 Free PMC article. Review.
The aim of this review is to summarize the evidence on efficacy, effectiveness and safety of intravenous enzyme replacement therapy (ERT) available for mucopolysaccharidoses (MPSs) I, II, IVA, VI and VII, gained in phase III clinical trials and in observational post …
The aim of this review is to summarize the evidence on efficacy, effectiveness and safety of intravenous enzyme replacement therapy (ERT) av …
In Vivo Gene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon.
Marcó S, Haurigot V, Bosch F. Marcó S, et al. Hum Gene Ther. 2019 Oct;30(10):1211-1221. doi: 10.1089/hum.2019.217. Hum Gene Ther. 2019. PMID: 31482754 Review.
Gene therapy is an attractive tool for treatment of LSDs caused by deficiencies in secretable lysosomal enzymes, in which neither full restoration of normal enzymatic activity nor transduction of all cells of the affected organ is necessary. However, some LSDs, such as mucopol …
Gene therapy is an attractive tool for treatment of LSDs caused by deficiencies in secretable lysosomal enzymes, in which neither full resto …
Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA.
Bugiani M, Abbink TEM, Edridge AWD, van der Hoek L, Hillen AEJ, van Til NP, Hu-A-Ng GV, Breur M, Aiach K, Drevot P, Hocquemiller M, Laufer R, Wijburg FA, van der Knaap MS. Bugiani M, et al. Ann Clin Transl Neurol. 2023 Jun;10(6):904-917. doi: 10.1002/acn3.51772. Epub 2023 May 11. Ann Clin Transl Neurol. 2023. PMID: 37165777 Free PMC article.
OBJECTIVE: Mucopolysaccharidosis type IIIA (MPSIIIA) caused by recessive SGSH variants results in sulfamidase deficiency, leading to neurocognitive decline and death. No disease-modifying therapy is available. The AAVance gene therapy trial investigates AAVrh.10 ove …
OBJECTIVE: Mucopolysaccharidosis type IIIA (MPSIIIA) caused by recessive SGSH variants results in sulfamidase deficiency, leading to …
159 results