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]

• Cross-matching ID: TTD ID: D00NYL

References

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