Sevin C - Search Results

1

A multicenter open-label extension study of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A.

Wijburg FA, Whitley CB, Muenzer J, Gasperini S, Del Toro M, Muschol N, Cleary M, Sevin C, Shapiro E, Alexanderian D. Wijburg FA, et al. Among authors: sevin c. Mol Genet Metab. 2021 Sep-Oct;134(1-2):175-181. doi: 10.1016/j.ymgme.2021.07.001. Epub 2021 Jul 7. Mol Genet Metab. 2021. PMID: 34247932 Free article. Clinical Trial.

2

Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries.

Schoenmakers DH, Mochel F, Adang LA, Boelens JJ, Calbi V, Eklund EA, Grønborg SW, Fumagalli F, Groeschel S, Lindemans C, Sevin C, Schöls L, Ram D, Zerem A, Graessner H, Wolf NI. Schoenmakers DH, et al. Among authors: sevin c. Orphanet J Rare Dis. 2024 Feb 7;19(1):46. doi: 10.1186/s13023-024-03075-3. Orphanet J Rare Dis. 2024. PMID: 38326898 Free PMC article.

3

Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States.

Adang LA, Bonkowsky JL, Boelens JJ, Mallack E, Ahrens-Nicklas R, Bernat JA, Bley A, Burton B, Darling A, Eichler F, Eklund E, Emrick L, Escolar M, Fatemi A, Fraser JL, Gaviglio A, Keller S, Patterson MC, Orchard P, Orthmann-Murphy J, Santoro JD, Schöls L, Sevin C, Srivastava IN, Rajan D, Rubin JP, Van Haren K, Wasserstein M, Zerem A, Fumagalli F, Laugwitz L, Vanderver A. Adang LA, et al. Among authors: sevin c. Cytotherapy. 2024 Jul;26(7):739-748. doi: 10.1016/j.jcyt.2024.03.487. Epub 2024 Apr 1. Cytotherapy. 2024. PMID: 38613540 Review.

4

Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial.

Wijburg FA, Whitley CB, Muenzer J, Gasperini S, Del Toro M, Muschol N, Cleary M, Sevin C, Shapiro E, Bhargava P, Kerr D, Alexanderian D. Wijburg FA, et al. Among authors: sevin c. Mol Genet Metab. 2019 Feb;126(2):121-130. doi: 10.1016/j.ymgme.2018.10.006. Epub 2018 Oct 24. Mol Genet Metab. 2019. PMID: 30528227 Free article. Clinical Trial.

5

Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial.

Í Dali C, Sevin C, Krägeloh-Mann I, Giugliani R, Sakai N, Wu J, Wasilewski M. Í Dali C, et al. Among authors: sevin c. Mol Genet Metab. 2020 Sep-Oct;131(1-2):235-244. doi: 10.1016/j.ymgme.2020.07.002. Epub 2020 Jul 16. Mol Genet Metab. 2020. PMID: 32792226 Free article. Clinical Trial.

6

Adenosine kinase deficiency: Three new cases and diagnostic value of hypermethioninemia.

Becker PH, Demir Z, Mozer Glassberg Y, Sevin C, Habes D, Imbard A, Mussini C, Rozenfeld Bar Lev M, Davit-Spraul A, Benoist JF, Thérond P, Slama A, Jacquemin E, Gonzales E, Gaignard P. Becker PH, et al. Among authors: sevin c. Mol Genet Metab. 2021 Jan;132(1):38-43. doi: 10.1016/j.ymgme.2020.11.007. Epub 2020 Nov 28. Mol Genet Metab. 2021. PMID: 33309011

7

Clinical Trials for Gene Therapy in Lysosomal Diseases With CNS Involvement.

Sevin C, Deiva K. Sevin C, et al. Front Mol Biosci. 2021 Sep 16;8:624988. doi: 10.3389/fmolb.2021.624988. eCollection 2021. Front Mol Biosci. 2021. PMID: 34604300 Free PMC article. Review.

8

Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate.

Colle MA, Piguet F, Bertrand L, Raoul S, Bieche I, Dubreil L, Sloothaak D, Bouquet C, Moullier P, Aubourg P, Cherel Y, Cartier N, Sevin C. Colle MA, et al. Among authors: sevin c. Hum Mol Genet. 2010 Jan 1;19(1):147-58. doi: 10.1093/hmg/ddp475. Hum Mol Genet. 2010. PMID: 19837699

9

Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy.

Sevin C, Benraiss A, Van Dam D, Bonnin D, Nagels G, Verot L, Laurendeau I, Vidaud M, Gieselmann V, Vanier M, De Deyn PP, Aubourg P, Cartier N. Sevin C, et al. Hum Mol Genet. 2006 Jan 1;15(1):53-64. doi: 10.1093/hmg/ddi425. Epub 2005 Nov 25. Hum Mol Genet. 2006. PMID: 16311251

10

Enzyme, cell and gene-based therapies for metachromatic leukodystrophy.

Sevin C, Aubourg P, Cartier N. Sevin C, et al. J Inherit Metab Dis. 2007 Apr;30(2):175-83. doi: 10.1007/s10545-007-0540-z. Epub 2007 Mar 8. J Inherit Metab Dis. 2007. PMID: 17347913 Review.

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