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Results: 1 to 20 of 115

Similar articles for PubMed (Select 23858624)

1.

Skeletal abnormalities in lysosomal storage diseases.

Stevenson DA, Steiner RD.

Pediatr Endocrinol Rev. 2013 Jun;10 Suppl 2:406-16. Review.

PMID:
23858624
2.

Astrocytes and lysosomal storage diseases.

Rama Rao KV, Kielian T.

Neuroscience. 2015 May 30. pii: S0306-4522(15)00503-5. doi: 10.1016/j.neuroscience.2015.05.061. [Epub ahead of print] Review.

PMID:
26037807
3.

The clinical spectrum and pathophysiology of skeletal complications in lysosomal storage disorders.

Clarke LA, Hollak CE.

Best Pract Res Clin Endocrinol Metab. 2015 Mar;29(2):219-235. doi: 10.1016/j.beem.2014.08.010. Epub 2014 Aug 27. Review.

PMID:
25987175
4.

The role of antibodies in enzyme treatments and therapeutic strategies.

Bigger BW, Saif M, Linthorst GE.

Best Pract Res Clin Endocrinol Metab. 2015 Mar;29(2):183-194. doi: 10.1016/j.beem.2015.01.006. Epub 2015 Jan 24. Review.

PMID:
25987172
5.

Non-neuronopathic lysosomal storage disorders: Disease spectrum and treatments.

Pastores GM, Hughes DA.

Best Pract Res Clin Endocrinol Metab. 2015 Mar;29(2):173-182. doi: 10.1016/j.beem.2014.08.005. Epub 2014 Aug 27. Review.

PMID:
25987171
6.

Epidemiology and diagnosis of lysosomal storage disorders; challenges of screening.

Kingma SD, Bodamer OA, Wijburg FA.

Best Pract Res Clin Endocrinol Metab. 2015 Mar;29(2):145-157. doi: 10.1016/j.beem.2014.08.004. Epub 2014 Aug 26. Review.

PMID:
25987169
7.

Mechanistic and therapeutic insights gained from studying rare skeletal diseases.

Tosi LL, Warman ML.

Bone. 2015 Jul;76:67-75. doi: 10.1016/j.bone.2015.03.016. Epub 2015 Mar 27. Review.

8.

A review of gene therapy in canine and feline models of lysosomal storage disorders.

Bradbury AM, Gurda BL, Casal ML, Ponder KP, Vite CH, Haskins ME.

Hum Gene Ther Clin Dev. 2015 Mar;26(1):27-37. doi: 10.1089/humc.2015.002. Epub 2015 Feb 11.

PMID:
25671613
9.

A review of gene therapy in canine and feline models of lysosomal storage disorders.

Bradbury AM, Gurda BL, Casal ML, Ponder K, Vite C, Haskins ME.

Hum Gene Ther Clin Dev. 2015 Jan 14. [Epub ahead of print]

PMID:
25587775
10.

Treatment for Lsds: real options for several diseases. Forward.

Cohen IJ, Baris HN, Mistry PK, Sands MS.

Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:71. No abstract available.

PMID:
25345087
11.

From bedside to cell biology: a century of history on lysosomal dysfunction.

Coutinho MF, Matos L, Alves S.

Gene. 2015 Jan 15;555(1):50-8. doi: 10.1016/j.gene.2014.09.054. Epub 2014 Sep 29. Review.

PMID:
25275857
12.

Clinical studies in lysosomal storage diseases: Past, present, and future.

Boudes PF.

Rare Dis. 2013 Oct 7;1:e26690. doi: 10.4161/rdis.26690. eCollection 2013. Review.

13.

Lysosomal storage diseases: Stem cell-based cell- and gene-therapy.

Kim SU.

Cell Transplant. 2014 May 21. [Epub ahead of print]

PMID:
24853878
14.

Similar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage disease.

Ferla R, Claudiani P, Cotugno G, Saccone P, De Leonibus E, Auricchio A.

Hum Gene Ther. 2014 Jul;25(7):609-18. doi: 10.1089/hum.2013.213. Epub 2014 Apr 11.

15.

The NCS-LSD cohort study: a description of the methods and analyses used to assess the long-term effectiveness of enzyme replacement therapy and substrate reduction therapy in patients with lysosomal storage disorders.

Henley WE, Anderson LJ, Wyatt KM, Nikolaou V, Anderson R, Logan S.

J Inherit Metab Dis. 2014 Nov;37(6):939-44. doi: 10.1007/s10545-014-9679-6. Epub 2014 Feb 12.

PMID:
24519353
16.

Treatment of lysosomal storage diseases: recent patents and future strategies.

Ortolano S, ViƩitez I, Navarro C, Spuch C.

Recent Pat Endocr Metab Immune Drug Discov. 2014 Jan;8(1):9-25. Review.

PMID:
24433521
17.

Hematopoietic stem cell transplant for lysosomal storage diseases.

Lund TC.

Pediatr Endocrinol Rev. 2013 Nov;11 Suppl 1:91-8. Review.

PMID:
24380127
18.

Oral small molecule therapy for lysosomal storage diseases.

Weinreb NJ.

Pediatr Endocrinol Rev. 2013 Nov;11 Suppl 1:77-90. Review.

PMID:
24380126
19.

Clinical studies in lysosomal storage diseases: past, present and future.

Boudes PF.

Pediatr Endocrinol Rev. 2013 Nov;11 Suppl 1:68-76. Review.

PMID:
24380125
20.

Treatment for LSDs: no longer just enzyme replacement therapy for Gaucher disease. Foreword.

Cohen IJ, Baris H, Mistry PK.

Pediatr Endocrinol Rev. 2013 Nov;11 Suppl 1:58. No abstract available.

PMID:
24380122
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