ITVISMA (onasemnogene abeparvovec-brve)
GENE THERAPY AGENTS - SMN PROTEIN DEFICIENCY · SPINAL MUSCULAR ATROPHY
AVEXIS, INC.
Drug class
Gene Therapy Agents - SMN Protein Deficiency
Gene Therapy Agents - SMN Protein Deficiency is a specialized formulary and administrative drug classification rather than a broad, multi-drug pharmacological class. Read more about Gene Therapy Agents - SMN Protein Deficiency
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Gene Therapy Agents - SMN Protein Deficiency is a specialized formulary and administrative drug classification rather than a broad, multi-drug pharmacological class.
Established in standard compendia primarily to categorize adeno-associated virus-based gene replacement therapies such as onasemnogene abeparvovec, these agents function by delivering a functional copy of the human *SMN1* gene directly into target motor neurons.
By restoring sustained expression of the survival motor neuron (SMN) protein, the therapy addresses the root genetic cause of neuromuscular degeneration.
Consequently, this class is utilized exclusively for the management of spinal muscular atrophy (SMA) associated with bi-allelic *SMN1* mutations.
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ncdhhs.gov
Gene Therapy Agents - SMN Protein Deficiency is a specialized formulary and administrative drug classification rather than a broad, multi-drug pharmacological class Established in standard compendia primarily to categorize adeno-associated virus-based gene replacement therapies such as onasemnogene abeparvovec, these agents function by delivering a functional copy of the human *SMN1* gene directly into target motor neurons
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la.gov
Gene Therapy Agents - SMN Protein Deficiency is a specialized formulary and administrative drug classification rather than a broad, multi-drug pharmacological class
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fda.gov
Established in standard compendia primarily to categorize adeno-associated virus-based gene replacement therapies such as onasemnogene abeparvovec, these agents function by delivering a functional copy of the human *SMN1* gene directly into target motor neurons By restoring sustained expression of the survival motor neuron (SMN) protein, the therapy addresses the root genetic cause of neuromuscular degeneration Consequently, this class is utilized exclusively for the management of spinal muscula...
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drugbank.com
By restoring sustained expression of the survival motor neuron (SMN) protein, the therapy addresses the root genetic cause of neuromuscular degeneration Consequently, this class is utilized exclusively for the management of spinal muscular atrophy (SMA) associated with bi-allelic *SMN1* mutations
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