Is gene therapy in spinal muscular atrophy safe? A casereport of thrombotic microangiopathy following onasemnogene abeparvovec
Spinal muscular atrophy is a neuromuscular disorder caused by a mutation in the survival of SMN1 gene. Diagnosis of the disease is based mainly on the presence of hypotonia and symmetrical [1]. A five-month-old male with SMA type 1 was admitted to the Children’s Neurology Clinic for gene therapy wit...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Institute of Rural Health
2022-10-01
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| Series: | Journal of Pre-Clinical and Clinical Research |
| Subjects: | |
| Online Access: | https://www.jpccr.eu/Is-gene-therapy-in-spinal-muscular-atrophy-safe-A-casereport-of-thrombotic-microangiopathy,154984,0,2.html |