Co-creating a gene therapy clinical trial with GM2 gangliosidosis caregivers: A virtual approach to patient engagement

2021 ◽  
Vol 132 (2) ◽  
pp. S61
Author(s):  
Kristin LaBounty Phillips ◽  
Emily McGinnis ◽  
Jennifer Seda ◽  
Kimberly Goodspeed ◽  
Suyesh Prasad
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Patient Engagement ◽  
Gm2 Gangliosidosis ◽  
Therapy Clinical Trial

scholarly journals Evaluation of Leberʼs hereditary optic neuropathy patients prior to a gene therapy clinical trial

Medicine ◽  
2016 ◽  
Vol 95 (40) ◽  
pp. e5110 ◽  
Author(s):  
Shuo Yang ◽  
Hong Yang ◽  
Si-qi Ma ◽  
Shuai-shuai Wang ◽  
Heng He ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Optic Neuropathy ◽  
Therapy Clinical Trial ◽  
Hereditary Optic Neuropathy

AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: decreased need of ventilatory and nutritional support at End-of-Study

2017 ◽  
Vol 27 ◽  
pp. S208
Author(s):  
R. Shell ◽  
S. Al-Zaidy ◽  
W. Arnold ◽  
L. Rodino-Klapac ◽  
T. Prior ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Nutritional Support ◽  
Phase 1 ◽  
Therapy Clinical Trial

scholarly journals Leber Congenital Amaurosis gene therapy clinical trial

2009 ◽  
Vol 9 (14) ◽  
pp. 27-27 ◽  
Author(s):  
W. W. Hauswirth ◽  
A. V. Cideciyan ◽  
T. S. Aleman ◽  
S. Kaushal ◽  
B. J. Byrne ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Leber Congenital Amaurosis ◽  
Therapy Clinical Trial

scholarly journals The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup

2020 ◽  
Vol 31 (19-20) ◽  
pp. 1034-1042
Author(s):  
Philip J. Brooks ◽  
Elizabeth A. Ottinger ◽  
Deanna Portero ◽  
Richa Madan Lomash ◽  
Asaf Alimardanov ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Adeno Associated Virus ◽  
Virus Gene ◽  
Gene Therapies ◽  
Therapy Clinical Trial

AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Interim data demonstrates improvements in supportive care use

2017 ◽  
Vol 21 ◽  
pp. e14 ◽  
Author(s):  
Richard Shell ◽  
Samiah Al-Zaidy ◽  
W. Dave Arnold ◽  
Louise Rodino-Klapac ◽  
Thomas W. Prior ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Supportive Care ◽  
Phase 1 ◽  
Therapy Clinical Trial
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