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
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