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Get to know ITVISMA.

Consider the potential of one-time gene replacement therapy for your patients and practice

Learn the steps to initiating ITVISMA therapy

AAV9, adeno-associated virus serotype 9; ALT, alanine aminotransferase; AST, aspartate aminotransferase; DNA, deoxyribonucleic acid; MOA, mechanism of action; SMA, spinal muscular atrophy; SMN, survival motor neuron; SMN, survival motor neuron gene; SMN1, survival motor neuron 1 gene.
References: 1. ITVISMA. Prescribing information. Novartis Gene Therapies, Inc. 2. SPINRAZA. Prescribing information. Biogen. 3. EVRYSDI. Prescribing information. Genentech, Inc. 4. Burghes AHM, Beattie CE. Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick? Nat Rev Neurosci. 2009;10(8):597-609. doi:10.1038/nrn2670 5. McGovern VL, Iyer CC, Arnold WD, et al. SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA. Hum Mol Genet. 2015;24(19):5524-5541. doi:10.1093/hmg/ddv283 6. Coratti G, Bovis F, Pera MC, et al; ISMAC group. Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: an international study. Eur J Neurol. 2024;31(8):e16309. doi:10.1111/ene.16309 7. Colella P, Ronzitti G, Mingozzi F. Emerging issues in AAV-mediated in vivo gene therapy. Mol Ther Methods Clin Dev. 2017;8:87-104. doi:10.1016/j.omtm.2017.11.007 8. Haggerty DL, Grecco GG, Reeves KC, Atwood B. Adeno-associated viral vectors in neuroscience research. Mol Ther Methods Clin Dev. 2019;17:69-82. doi:10.1016/j.omtm.2019.11.012 9. Mendell JR, AI-Zaidy S, Shell R, et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med. 2017;377(18):1713-1722. doi:10.1056/NEJMoa1706198