Details of the Drug

General Information of Drug (ID: DMBZS2G)

Drug Name

AAV2/8-CBA-hSMN1

Synonyms

ScAAV2/8-GUSB-hSMN1; SMN1 gene therapy (AAV8-serotype vector, spinal muscular atrophy), Genzyme; Survival motor neuron 1 gene therapy (adeno-associated viral vector, spinal muscular atrophy), Genzyme

Indication

Disease Entry	ICD 11	Status	REF
Spinal muscular atrophy	8B61	Investigative	[1]
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Cross-matching ID

TTD ID: D00NYL

References

1	The ChEMBL database in 2017. Nucleic Acids Res. 2017 Jan 4;45(D1):D945-D954.