Thrombotic Microangiopathy (TMA) after Gene Replacement Therapy (GRT) Due to Spinal Muscular Atrophy: Case Summary and Recommendations for Treatment

2021 ◽  
Author(s):  
D. Witte ◽  
H. Hartmann ◽  
J. Drube ◽  
D. Haffner ◽  
S. Illsinger
Keyword(s):  
Spinal Muscular Atrophy ◽  
Replacement Therapy ◽  
Thrombotic Microangiopathy ◽  
Muscular Atrophy ◽  
Gene Replacement ◽  
Case Summary ◽  
Gene Replacement Therapy

scholarly journals European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy

2020 ◽  
Vol 28 ◽  
pp. 38-43 ◽  
Author(s):  
Janbernd Kirschner ◽  
Nina Butoianu ◽  
Nathalie Goemans ◽  
Jana Haberlova ◽  
Anna Kostera-Pruszczyk ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Replacement Therapy ◽  
Ad Hoc ◽  
Consensus Statement ◽  
Muscular Atrophy ◽  
Gene Replacement ◽  
Gene Replacement Therapy

P.350Onasemnogene abeparvovec gene-replacement therapy (GRT) in pre-symptomatic spinal muscular atrophy (SMA)

2019 ◽  
Vol 29 ◽  
pp. S183
Author(s):  
M. Schultz ◽  
K. Swoboda ◽  
M. Farrar ◽  
H. McMillan ◽  
J. Parsons ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Replacement Therapy ◽  
Muscular Atrophy ◽  
Gene Replacement ◽  
Gene Replacement Therapy

scholarly journals Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

2017 ◽  
Vol 377 (18) ◽  
pp. 1713-1722 ◽  
Author(s):  
Jerry R. Mendell ◽  
Samiah Al-Zaidy ◽  
Richard Shell ◽  
W. Dave Arnold ◽  
Louise R. Rodino-Klapac ◽  
...  
Keyword(s):  
Single Dose ◽  
Spinal Muscular Atrophy ◽  
Replacement Therapy ◽  
Muscular Atrophy ◽  
Gene Replacement ◽  
Gene Replacement Therapy
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