Ries M - Search Results

1

Quantitative clinical characteristics of 53 patients with MPS VII: a cross-sectional analysis.

Zielonka M, Garbade SF, Kölker S, Hoffmann GF, Ries M. Zielonka M, et al. Among authors: ries m. Genet Med. 2017 Sep;19(9):983-988. doi: 10.1038/gim.2017.10. Epub 2017 Apr 6. Genet Med. 2017. PMID: 28383542 Free article.

2

The relationship of vascular glycolipid storage to clinical manifestations of Fabry disease: a cross-sectional study of a large cohort of clinically affected heterozygous women.

Gupta S, Ries M, Kotsopoulos S, Schiffmann R. Gupta S, et al. Among authors: ries m. Medicine (Baltimore). 2005 Sep;84(5):261-268. doi: 10.1097/01.md.0000178976.62537.6b. Medicine (Baltimore). 2005. PMID: 16148726 Free article.

3

Quantitative dysmorphology assessment in Fabry disease.

Ries M, Moore DF, Robinson CJ, Tifft CJ, Rosenbaum KN, Brady RO, Schiffmann R, Krasnewich D. Ries M, et al. Genet Med. 2006 Feb;8(2):96-101. doi: 10.1097/01.gim.0000200950.25118.dd. Genet Med. 2006. PMID: 16481892 Free article.

4

Changes in plasma and urine globotriaosylceramide levels do not predict Fabry disease progression over 1 year of agalsidase alfa.

Schiffmann R, Ries M, Blankenship D, Nicholls K, Mehta A, Clarke JT, Steiner RD, Beck M, Barshop BA, Rhead W, West M, Martin R, Amato D, Nair N, Huertas P. Schiffmann R, et al. Among authors: ries m. Genet Med. 2013 Dec;15(12):983-9. doi: 10.1038/gim.2013.56. Epub 2013 May 16. Genet Med. 2013. PMID: 23680766 Free article. Clinical Trial.

5

Ultra-orphan diseases: a quantitative analysis of the natural history of molybdenum cofactor deficiency.

Mechler K, Mountford WK, Hoffmann GF, Ries M. Mechler K, et al. Among authors: ries m. Genet Med. 2015 Dec;17(12):965-70. doi: 10.1038/gim.2015.12. Epub 2015 Mar 12. Genet Med. 2015. PMID: 25764214 Free article.

6

Pressure for drug development in lysosomal storage disorders - a quantitative analysis thirty years beyond the US orphan drug act.

Mechler K, Mountford WK, Hoffmann GF, Ries M. Mechler K, et al. Among authors: ries m. Orphanet J Rare Dis. 2015 Apr 18;10:46. doi: 10.1186/s13023-015-0262-5. Orphanet J Rare Dis. 2015. PMID: 25896727 Free PMC article.

7

Clinical Research in Vulnerable Populations: Variability and Focus of Institutional Review Boards' Responses.

Kästner B, Behre S, Lutz N, Bürger F, Luntz S, Hinderhofer K, Bendszus M, Hoffmann GF, Ries M. Kästner B, et al. Among authors: ries m. PLoS One. 2015 Aug 14;10(8):e0135997. doi: 10.1371/journal.pone.0135997. eCollection 2015. PLoS One. 2015. PMID: 26275228 Free PMC article. Clinical Trial.

8

Ten Years after the International Committee of Medical Journal Editors' Clinical Trial Registration Initiative, One Quarter of Phase 3 Pediatric Epilepsy Clinical Trials Still Remain Unpublished: A Cross Sectional Analysis.

Lampert A, Hoffmann GF, Ries M. Lampert A, et al. Among authors: ries m. PLoS One. 2016 Jan 6;11(1):e0144973. doi: 10.1371/journal.pone.0144973. eCollection 2016. PLoS One. 2016. PMID: 26735955 Free PMC article.

9

Novel Treatments for Rare Cancers: The U.S. Orphan Drug Act Is Delivering-A Cross-Sectional Analysis.

Stockklausner C, Lampert A, Hoffmann GF, Ries M. Stockklausner C, et al. Among authors: ries m. Oncologist. 2016 Apr;21(4):487-93. doi: 10.1634/theoncologist.2015-0397. Epub 2016 Mar 28. Oncologist. 2016. PMID: 27022038 Free PMC article.

10

Novel treatments for rare rheumatologic disorders: analysis of the impact of 30 years of the US orphan drug act.

Lutz T, Lampert A, Hoffmann GF, Ries M. Lutz T, et al. Among authors: ries m. Orphanet J Rare Dis. 2016 May 12;11(1):60. doi: 10.1186/s13023-016-0443-x. Orphanet J Rare Dis. 2016. PMID: 27176041 Free PMC article.

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