ZOLGENSMA (onasemnogene abeparvovec-xioi) suspension

Zolgensma is a gene therapy medicine for treating spinal muscular atrophy, a serious condition of the nerves that causes muscle wasting and weakness. Brand Name: ZOLGENSMA (onasemnogene abeparvovec-xioi) suspension, for intravenous infusion Initial U.S. Approval: 2019

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ZOLGENSMA (onasemnogene abeparvovec-xioi) suspension Price In India
ZOLGENSMA (onasemnogene abeparvovec-xioi) suspension, for intravenous infusion

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ZOLGENSMA (onasemnogene abeparvovec-xioi) suspension, for intravenous infusion

Onasemnogene abeparvovec-xioi injection is used to treat spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

ZOLGENSMA is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Limitations of Use
• The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated.
• The use of ZOLGENSMA in patients with advanced SMA (e.g.,complete paralysis of limbs, permanent ventilator dependence) has not been evaluated.

Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. It works by providing a new copy of the SMN gene that produces the SMN protein.

In the United States, onasemnogene abeparvovec is indicated for the treatment of people less than two years of age with spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

The treatment is approved in the United States and certain other countries for use in children with spinal muscular atrophy up to the age of two, including at the presymptomatic stage of the disease. In the European Union and Canada, it is indicated for the treatment of people with spinal muscular atrophy who either have a clinical diagnosis of spinal muscular atrophy type 1 or have up to three copies of the SMN2 gene.

Drug (Brand / Generic): ZOLGENSMA / onasemnogene abeparvovec-xioi
Current Indications: spinal muscular atrophy (SMA)
Marketed by:: Novartis Gene Therapies
Approval Date: 2019

ZOLGENSMA is a suspension for intravenous infusion, supplied as single-use vials.
ZOLGENSMA is provided in a kit containing 2 to 14 vials, as a combination 2 vial fill volumes (either 5.5 mL or 8.3 mL). All vials have a nominal
concentration of 2.0 × 1013 vector genomes (vg) per mL. Each vial of OLGENSMA contains an extractable volume of not less than either 5.5 mL or
8.3 mL.

For more highlights of prescribing information regarding Indications and usage, dosage and administration, dosage forms and strengths, patient counseling information and medication guide. Click Here

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