80 The set up and running of the first gene therapy trial for patients with SMA in the clinical research facility at great ormond street hospital

2021 ◽  
Author(s):  
Elizabeth Vella ◽  
Allyson Gray ◽  
Katie Groves
Keyword(s):  
Gene Therapy ◽  
Clinical Research ◽  
Research Facility ◽  
Street Hospital ◽  
Gene Therapy Trial ◽  
Great Ormond Street Hospital ◽  
Clinical Research Facility ◽  
Therapy Trial ◽  
Set Up

ASPIRO Gene Therapy Trial in X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Efficacy and Safety Findings

2021 ◽  
Author(s):  
A. Blaschek ◽  
P. B. Shieh ◽  
N. Kuntz ◽  
J. J. Dowling ◽  
W. Müller-Felber ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Efficacy And Safety ◽  
Myotubular Myopathy ◽  
Gene Therapy Trial ◽  
Therapy Trial
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