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IP 853. AVXS-101 Phase-1-Gene Therapy Clinical Trial in SMA Type 1: Event-Free Survival and Achievement of Developmental Milestones
Mapping Intimacies
◽
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
Related Documents
Cited By
References
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
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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: 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
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
AVXS-101 Phase 1 Gene-Replacement Therapy (GRT) Clinical Trial in Spinal Muscular Atrophy Type 1 (SMA1): 24-Month Event-Free Survival and Achievement of Developmental Milestones
10.1055/s-0039-1685425
◽
2019
◽
Cited By ~ 1
Author(s):
J.R. Mendell
◽
S. Al-Zaidy
◽
R. Shell
◽
W.D. Arnold
◽
L. Rodino-Klapac
◽
...
Keyword(s):
Clinical Trial
◽
Spinal Muscular Atrophy
◽
Muscular Atrophy
◽
Phase 1
◽
Gene Replacement
◽
Event Free Survival
◽
Free Survival
◽
Spinal Muscular Atrophy Type
◽
Gene Replacement Therapy
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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):
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◽
Gene Therapy
◽
Therapeutic Dose
◽
Phase 1
◽
Younger Age
◽
Therapy Clinical Trial
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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
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Jerry R. Mendell
◽
Richard Shell
◽
Kelly J. Lehman
◽
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◽
Linda P. Lowes
◽
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◽
Muscular Atrophy
◽
Phase 1
◽
Gene Replacement
◽
Spinal Muscular Atrophy Type
◽
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◽
Long Term Follow Up
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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
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Gaucher Disease
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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
◽
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Heng He
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Clinical Trial
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Gene Therapy
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◽
Therapy Clinical Trial
◽
Hereditary Optic Neuropathy
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Faculty Opinions recommendation of Effect of Blinatumomab vs Chemotherapy on Event-Free Survival Among Children With High-risk First-Relapse B-Cell Acute Lymphoblastic Leukemia: A Randomized Clinical Trial.
Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
◽
10.3410/f.739661898.793584166
◽
2021
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Author(s):
Ching-Hon Pui
Keyword(s):
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High Risk
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B Cell
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Randomized Clinical Trial
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Lymphoblastic Leukemia
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Event Free Survival
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Cell Acute Lymphoblastic Leukemia
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