The Medical Letter on Drugs and Therapeutics
FROM
ISSUE 1577
ISSUE 1577
July 29, 2019
Issue 1577
- Zolgensma - One-Time Gene Therapy for Spinal Muscular Atrophy
- Bremelanotide (Vyleesi) for Hypoactive Sexual Desire Disorder
- Cyclosporine 0.09% Solution (Cequa) for Dry Eye Disease
- Cladribine (Mavenclad) for Multiple Sclerosis
- Addendum: Estradiol/Progesterone (Bijuva) for Menopausal Vasomotor Symptoms
- Correction: Plenvu
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Zolgensma - One-Time Gene Therapy for Spinal Muscular Atrophy
July 29, 2019 (Issue: 1577)
The FDA has approved onasemnogene abeparvovec-xioi (Zolgensma – Avexis), an adeno-associated virus
vector-based gene therapy, for one-time IV treatment
of children <2 years old who have spinal muscular
atrophy (SMA) and bi-allelic mutations in the survival
motor neuron 1 (SMN1) gene. It is the first gene therapy
and the second drug to be approved in the US for SMA;
nusinersen (Spinraza) was approved in 2016.
... more
- Nusinersen (Spinraza) for spinal muscular atrophy. Med Lett Drugs Ther 2017; 59:50.
- EA Sugarman et al. Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens. Eur J Hum Genet 2012; 20:27.
- SJ Kolb et al. Natural history of infantile-onset spinal muscular atrophy. Ann Neurol 2017; 82:883.
- MA Waldrop and SJ Kolb. Current treatment options in neurology-SMA therapeutics. Curr Treat Options Neurol 2019; 21:25.
- SJ Kolb and JT Kissel. Spinal muscular atrophy. Neurol Clin 2015; 33:831.
- TW Prior et al. Newborn and carrier screening for spinal muscular atrophy. Am J Med Genet A 2010; 152A:1608.
- RS Finkel et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med 2017; 377:1723.
- E Mercuri et al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med 2018; 378:625.
- FDA summary basis for regulatory action. Available at: www.fda.gov. Accessed July 18, 2019.
- JR Mendell et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med 2017; 377:1713.

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