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Seminars in Hematology
|
October 7, 2004
Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients
Neal J Weinreb, Mario C Aggio, Hans C Andersson, et al.
Orphanet Journal of Rare Diseases
|
November 7, 2016
Increased Wnt and Notch signaling: a clue to the renal disease in Schimke immuno-osseous dysplasia?
Marie Morimoto, Clara Myung, Kimberly Beirnes, et al.
Lancet (London, England)
|
September 6, 2020
Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial
Ravi Savarirayan, Louise Tofts, Melita Irving, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
August 3, 2021
Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study
Ravi Savarirayan, Louise Tofts, Melita Irving, et al.
The New England Journal of Medicine
|
August 11, 2016
Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat
Dominique P Germain, Derralynn A Hughes, Kathleen Nicholls, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
September 15, 2022
Growth parameters in children with achondroplasia: A 7-year, prospective, multinational, observational study
Ravi Savarirayan, Melita Irving, Paul Harmatz, et al.
Orphanet Journal of Rare Diseases
|
September 25, 2012
Reduced elastogenesis: a clue to the arteriosclerosis and emphysematous changes in Schimke immuno-osseous dysplasia?
Marie Morimoto, Zhongxin Yu, Peter Stenzel, et al.
American Journal of Human Genetics
|
May 7, 2021
Variants in the degron of AFF3 are associated with intellectual disability, mesomelic dysplasia, horseshoe kidney, and epileptic encephalopathy
Norine Voisin, Rhonda E Schnur, Sofia Douzgou, et al.
American Journal of Human Genetics
|
August 4, 2015
Mutations in DDX3X Are a Common Cause of Unexplained Intellectual Disability with Gender-Specific Effects on Wnt Signaling
Lot Snijders Blok, Erik Madsen, Jane Juusola, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 8, 2020
Phenotypic spectrum and transcriptomic profile associated with germline variants in TRAF7
Laura Castilla-Vallmanya, Kaja K Selmer, Clémantine Dimartino, et al.
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Search research articles
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Showing results (101-110 of 110) with videos related to
Sort By:
Page
of 11
You have reached the last page of results.
This site can display upto 110 results.
Seminars in Hematology
|
October 7, 2004
Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients
Neal J Weinreb, Mario C Aggio, Hans C Andersson, et al.
Orphanet Journal of Rare Diseases
|
November 7, 2016
Increased Wnt and Notch signaling: a clue to the renal disease in Schimke immuno-osseous dysplasia?
Marie Morimoto, Clara Myung, Kimberly Beirnes, et al.
Lancet (London, England)
|
September 6, 2020
Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial
Ravi Savarirayan, Louise Tofts, Melita Irving, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
August 3, 2021
Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study
Ravi Savarirayan, Louise Tofts, Melita Irving, et al.
The New England Journal of Medicine
|
August 11, 2016
Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat
Dominique P Germain, Derralynn A Hughes, Kathleen Nicholls, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
September 15, 2022
Growth parameters in children with achondroplasia: A 7-year, prospective, multinational, observational study
Ravi Savarirayan, Melita Irving, Paul Harmatz, et al.
Orphanet Journal of Rare Diseases
|
September 25, 2012
Reduced elastogenesis: a clue to the arteriosclerosis and emphysematous changes in Schimke immuno-osseous dysplasia?
Marie Morimoto, Zhongxin Yu, Peter Stenzel, et al.
American Journal of Human Genetics
|
May 7, 2021
Variants in the degron of AFF3 are associated with intellectual disability, mesomelic dysplasia, horseshoe kidney, and epileptic encephalopathy
Norine Voisin, Rhonda E Schnur, Sofia Douzgou, et al.
American Journal of Human Genetics
|
August 4, 2015
Mutations in DDX3X Are a Common Cause of Unexplained Intellectual Disability with Gender-Specific Effects on Wnt Signaling
Lot Snijders Blok, Erik Madsen, Jane Juusola, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 8, 2020
Phenotypic spectrum and transcriptomic profile associated with germline variants in TRAF7
Laura Castilla-Vallmanya, Kaja K Selmer, Clémantine Dimartino, et al.
Page
of 11