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AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: decreased need of ventilatory and nutritional support at End-of-Study
Neuromuscular Disorders
◽
10.1016/j.nmd.2017.06.413
◽
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
Download Full-text
Related Documents
Cited By
References
AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Interim data demonstrates improvements in supportive care use
European Journal of Paediatric Neurology
◽
10.1016/j.ejpn.2017.04.1217
◽
2017
◽
Vol 21
◽
pp. e14
◽
Cited By ~ 1
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
Download Full-text
IP 853. AVXS-101 Phase-1-Gene Therapy Clinical Trial in SMA Type 1: Event-Free Survival and Achievement of Developmental Milestones
10.1055/s-0038-1676033
◽
2018
◽
Cited By ~ 1
Author(s):
Jerry Mendell
◽
Samiah Al-Zaidy
◽
Richard Shell
◽
W. Arnold
◽
Louise Rodino-Klapac
◽
...
Keyword(s):
Clinical Trial
◽
Gene Therapy
◽
Phase 1
◽
Developmental Milestones
◽
Event Free Survival
◽
Free Survival
◽
Therapy Clinical Trial
Download Full-text
AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones
European Journal of Paediatric Neurology
◽
10.1016/j.ejpn.2017.04.1216
◽
2017
◽
Vol 21
◽
pp. e13-e14
◽
Cited By ~ 2
Author(s):
Jerry R. Mendell
◽
Samiah Al-Zaidy
◽
Richard Shell
◽
W. David Arnold
◽
Louise Rodino-Klapac
◽
...
Keyword(s):
Clinical Trial
◽
Gene Therapy
◽
Phase 1
◽
Developmental Milestones
◽
Event Free Survival
◽
Free Survival
◽
Therapy Clinical Trial
Download Full-text
AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: end-of-Study event free survival and achievement of developmental milestones
Neuromuscular Disorders
◽
10.1016/j.nmd.2017.06.412
◽
2017
◽
Vol 27
◽
pp. S208
◽
Cited By ~ 4
Author(s):
J. Mendell
◽
S. Al-Zaidy
◽
R. Shell
◽
W. Arnold
◽
L. Rodino-Klapac
◽
...
Keyword(s):
Clinical Trial
◽
Gene Therapy
◽
Phase 1
◽
Developmental Milestones
◽
Event Free Survival
◽
Free Survival
◽
Therapy Clinical Trial
Download Full-text
AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: patients treated early with the proposed therapeutic dose were able to sit unassisted at a younger age
Neuromuscular Disorders
◽
10.1016/j.nmd.2017.06.414
◽
2017
◽
Vol 27
◽
pp. S208-S209
◽
Cited By ~ 1
Author(s):
L. Lowes
◽
S. Al-Zaidy
◽
R. Shell
◽
W. Arnold
◽
L. Rodino-Klapac
◽
...
Keyword(s):
Clinical Trial
◽
Gene Therapy
◽
Therapeutic Dose
◽
Phase 1
◽
Younger Age
◽
Therapy Clinical Trial
Download Full-text
Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2 Clinical Trial
10.1055/s-0039-1698259
◽
2019
◽
Cited By ~ 1
Author(s):
Jerry R. Mendell
◽
Richard Shell
◽
Kelly J. Lehman
◽
Markus McColly
◽
Linda P. Lowes
◽
...
Keyword(s):
Clinical Trial
◽
Muscular Atrophy
◽
Phase 1
◽
Gene Replacement
◽
Spinal Muscular Atrophy Type
◽
Gene Replacement Therapy
◽
Long Term Follow Up
Download Full-text
Phase 1/2 Lentiviral Vector Gene Therapy AVR-RD-02 for Subjects With Type 1 Gaucher Disease
Case Medical Research
◽
10.31525/ct1-nct04145037
◽
2019
◽
Author(s):
Keyword(s):
Gene Therapy
◽
Gaucher Disease
◽
Lentiviral Vector
◽
Phase 1
◽
Type 1 Gaucher Disease
Download Full-text
Evaluation of Leberʼs hereditary optic neuropathy patients prior to a gene therapy clinical trial
Medicine
◽
10.1097/md.0000000000005110
◽
2016
◽
Vol 95
(40)
◽
pp. e5110
◽
Cited By ~ 7
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
Download Full-text
706. Retrovirus Integration Site Distribution in Long-Term Gene-Corrected Hematopoiesis of the SCID-X1 Gene Therapy Clinical Trial
Molecular Therapy
◽
10.1016/j.ymthe.2004.06.615
◽
2004
◽
Vol 9
◽
pp. S269
Keyword(s):
Clinical Trial
◽
Gene Therapy
◽
Integration Site
◽
Site Distribution
◽
Retrovirus Integration
◽
Therapy Clinical Trial
Download Full-text
Interim results of Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA)
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2019.11.129
◽
2020
◽
Vol 129
(2)
◽
pp. S56-S57
◽
Cited By ~ 1
Author(s):
Kevin M. Flanigan
◽
Nicholas J.C. Smith
◽
Maria Luz Couce
◽
Kristen V. Truxal
◽
Kim L. McBride
◽
...
Keyword(s):
Clinical Trial
◽
Gene Therapy
◽
Single Dose
◽
Phase 1
◽
Type A
◽
Syndrome Type
◽
Mucopolysaccharidosis Type
◽
Sanfilippo Syndrome
◽
Type Iiia
◽
Mucopolysaccharidosis Type Iiia
Download Full-text
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