Thrombotic Microangiopathy (TMA) after Gene Replacemant Therapy (GRT) due to spinal muscular atrophy: case summary and recommendations for treatment
D Witte, H Hartmann, J Drube, D Haffner… - Klinische …, 2021 - europepmc.org
D Witte, H Hartmann, J Drube, D Haffner, S Illsinger
Klinische Padiatrie, 2021•europepmc.org5q-associated spinal muscular atrophy is one of the most severe and common genetic
diseases. In the last few years, innovative methods of therapy have been developed based
on SMN2 gene modification, such as splicing, or replacement of the damaged SMN1 gene
(gene replacement therapy, GRT). GRT is known to be accompanied by off target effects like
temporary elevation of liver and cardiac enzymes usually without serious clinical relevance.
We report a 4-year-old girl suffering from thrombotic microangiopathy (TMA) after GRT due …
diseases. In the last few years, innovative methods of therapy have been developed based
on SMN2 gene modification, such as splicing, or replacement of the damaged SMN1 gene
(gene replacement therapy, GRT). GRT is known to be accompanied by off target effects like
temporary elevation of liver and cardiac enzymes usually without serious clinical relevance.
We report a 4-year-old girl suffering from thrombotic microangiopathy (TMA) after GRT due …
5q-associated spinal muscular atrophy is one of the most severe and common genetic diseases. In the last few years, innovative methods of therapy have been developed based on SMN2 gene modification, such as splicing, or replacement of the damaged SMN1 gene (gene replacement therapy, GRT). GRT is known to be accompanied by off target effects like temporary elevation of liver and cardiac enzymes usually without serious clinical relevance. We report a 4-year-old girl suffering from thrombotic microangiopathy (TMA) after GRT due to 5q-SMA.
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