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Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
June 4, 2011
The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease
Suhrad G Banugaria, Sean N Prater, Yiu-Ki Ng, et al.
Journal of the Peripheral Nervous System : JPNS
|
February 7, 2023
Validation of the parent-proxy pediatric Charcot-Marie-Tooth disease quality of life outcome measure
Tong Tong Wu, Richard S Finkel, Carly E Siskind, et al.
Journal of the Peripheral Nervous System : JPNS
|
September 18, 2025
Designing and Implementing a Web-Based Platform for Accurate and Reliable Clinical Outcome Measures and Global Certification for Evaluating Charcot-Marie-Tooth disease
Kayla M D Cornett, Tim Estilow, Paula Bray, et al.
Plos One
|
December 19, 2013
Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy
Richard S Finkel, Kevin M Flanigan, Brenda Wong, et al.
Journal of Neuromuscular Diseases
|
November 11, 2025
Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registry
Yasemin Erbaş, Laurent Servais, Perry B Shieh, et al.
Med (New York, N.Y.)
|
March 11, 2023
Unusually severe muscular dystrophy upon in-frame deletion of the dystrophin rod domain and lack of compensation by membrane-localized utrophin
Svetlana Gorokhova, Joachim Schessl, Yaqun Zou, et al.
Drug Safety
|
August 12, 2021
Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy
John W Day, Jerry R Mendell, Eugenio Mercuri, et al.
JAMA
|
February 5, 1998
Comparison of paroxetine and nortriptyline in depressed patients with ischemic heart disease
S P Roose, F Laghrissi-Thode, J S Kennedy, et al.
Journal of the Peripheral Nervous System : JPNS
|
May 11, 2023
Validation of the parent-proxy version of the pediatric Charcot-Marie-Tooth disease quality of life instrument for children aged 0-7 years
Tong Tong Wu, Richard S Finkel, Carly E Siskind, et al.
Molecular Genetics and Metabolism
|
November 20, 2012
Juvenile-onset motor neuron disease caused by novel mutations in β-hexosaminidase
Tyler Mark Pierson, Paola A Torres, Bei-Jin Zeng, et al.
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Search research articles
Search
Showing results (151-160 of 289) with videos related to
Sort By:
Page
of 29
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
June 4, 2011
The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease
Suhrad G Banugaria, Sean N Prater, Yiu-Ki Ng, et al.
Journal of the Peripheral Nervous System : JPNS
|
February 7, 2023
Validation of the parent-proxy pediatric Charcot-Marie-Tooth disease quality of life outcome measure
Tong Tong Wu, Richard S Finkel, Carly E Siskind, et al.
Journal of the Peripheral Nervous System : JPNS
|
September 18, 2025
Designing and Implementing a Web-Based Platform for Accurate and Reliable Clinical Outcome Measures and Global Certification for Evaluating Charcot-Marie-Tooth disease
Kayla M D Cornett, Tim Estilow, Paula Bray, et al.
Plos One
|
December 19, 2013
Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy
Richard S Finkel, Kevin M Flanigan, Brenda Wong, et al.
Journal of Neuromuscular Diseases
|
November 11, 2025
Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registry
Yasemin Erbaş, Laurent Servais, Perry B Shieh, et al.
Med (New York, N.Y.)
|
March 11, 2023
Unusually severe muscular dystrophy upon in-frame deletion of the dystrophin rod domain and lack of compensation by membrane-localized utrophin
Svetlana Gorokhova, Joachim Schessl, Yaqun Zou, et al.
Drug Safety
|
August 12, 2021
Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy
John W Day, Jerry R Mendell, Eugenio Mercuri, et al.
JAMA
|
February 5, 1998
Comparison of paroxetine and nortriptyline in depressed patients with ischemic heart disease
S P Roose, F Laghrissi-Thode, J S Kennedy, et al.
Journal of the Peripheral Nervous System : JPNS
|
May 11, 2023
Validation of the parent-proxy version of the pediatric Charcot-Marie-Tooth disease quality of life instrument for children aged 0-7 years
Tong Tong Wu, Richard S Finkel, Carly E Siskind, et al.
Molecular Genetics and Metabolism
|
November 20, 2012
Juvenile-onset motor neuron disease caused by novel mutations in β-hexosaminidase
Tyler Mark Pierson, Paola A Torres, Bei-Jin Zeng, et al.
Page
of 29