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Nature Methods
|
March 31, 2018
Response to "Unexpected mutations after CRISPR-Cas9 editing in vivo"
Christopher J Wilson, Tim Fennell, Anne Bothmer, et al.
The Journal of Pediatrics
|
May 6, 2004
Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase)
James E Wraith, Lorne A Clarke, Michael Beck, et al.
American Journal of Human Genetics
|
May 7, 2002
Contiguous deletion of the X-linked adrenoleukodystrophy gene (ABCD1) and DXS1357E: a novel neonatal phenotype similar to peroxisomal biogenesis disorders
Deyanira Corzo, William Gibson, Kisha Johnson, et al.
Pharmacoepidemiology and Drug Safety
|
December 16, 2011
Source document verification in the Mucopolysaccharidosis Type I Registry
Karien Verhulst, Laura Artiles-Carloni, Michael Beck, et al.
Circulation. Heart Failure
|
February 15, 2017
Differences in Presentation and Outcomes Between Children With Familial Dilated Cardiomyopathy and Children With Idiopathic Dilated Cardiomyopathy: A Report From the Pediatric Cardiomyopathy Registry Study Group
Paolo Rusconi, James D Wilkinson, Lynn A Sleeper, et al.
Circulation
|
July 27, 2011
Competing risks for death and cardiac transplantation in children with dilated cardiomyopathy: results from the pediatric cardiomyopathy registry
Jorge A Alvarez, E John Orav, James D Wilkinson, et al.
Nature Genetics
|
July 16, 2002
Identification of the gene (BBS1) most commonly involved in Bardet-Biedl syndrome, a complex human obesity syndrome
Kirk Mykytyn, Darryl Y Nishimura, Charles C Searby, et al.
American Journal of Human Genetics
|
January 14, 2003
Evaluation of complex inheritance involving the most common Bardet-Biedl syndrome locus (BBS1)
Kirk Mykytyn, Darryl Y Nishimura, Charles C Searby, et al.
Blood Cells, Molecules & Diseases
|
April 28, 2019
Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1
Pramod K Mistry, Manisha Balwani, Hagit N Baris, et al.
Blood Cells, Molecules & Diseases
|
April 23, 2018
Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1
Pramod K Mistry, Manisha Balwani, Hagit N Baris, et al.
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Search research articles
Search
Showing results (61-70 of 75) with videos related to
Sort By:
Page
of 8
Nature Methods
|
March 31, 2018
Response to "Unexpected mutations after CRISPR-Cas9 editing in vivo"
Christopher J Wilson, Tim Fennell, Anne Bothmer, et al.
The Journal of Pediatrics
|
May 6, 2004
Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase)
James E Wraith, Lorne A Clarke, Michael Beck, et al.
American Journal of Human Genetics
|
May 7, 2002
Contiguous deletion of the X-linked adrenoleukodystrophy gene (ABCD1) and DXS1357E: a novel neonatal phenotype similar to peroxisomal biogenesis disorders
Deyanira Corzo, William Gibson, Kisha Johnson, et al.
Pharmacoepidemiology and Drug Safety
|
December 16, 2011
Source document verification in the Mucopolysaccharidosis Type I Registry
Karien Verhulst, Laura Artiles-Carloni, Michael Beck, et al.
Circulation. Heart Failure
|
February 15, 2017
Differences in Presentation and Outcomes Between Children With Familial Dilated Cardiomyopathy and Children With Idiopathic Dilated Cardiomyopathy: A Report From the Pediatric Cardiomyopathy Registry Study Group
Paolo Rusconi, James D Wilkinson, Lynn A Sleeper, et al.
Circulation
|
July 27, 2011
Competing risks for death and cardiac transplantation in children with dilated cardiomyopathy: results from the pediatric cardiomyopathy registry
Jorge A Alvarez, E John Orav, James D Wilkinson, et al.
Nature Genetics
|
July 16, 2002
Identification of the gene (BBS1) most commonly involved in Bardet-Biedl syndrome, a complex human obesity syndrome
Kirk Mykytyn, Darryl Y Nishimura, Charles C Searby, et al.
American Journal of Human Genetics
|
January 14, 2003
Evaluation of complex inheritance involving the most common Bardet-Biedl syndrome locus (BBS1)
Kirk Mykytyn, Darryl Y Nishimura, Charles C Searby, et al.
Blood Cells, Molecules & Diseases
|
April 28, 2019
Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1
Pramod K Mistry, Manisha Balwani, Hagit N Baris, et al.
Blood Cells, Molecules & Diseases
|
April 23, 2018
Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1
Pramod K Mistry, Manisha Balwani, Hagit N Baris, et al.
Page
of 8