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Generic Name: Onasemnogene abeparvovec-xioi
Dosage Form: Intravenous (infusion) injection
The class of Drug: Miscellaneous uncategorized agents

What is Zolgensma?

Zolgensma is a gene therapy that is used to treat the condition known as spinal muscular atrophy (SMA). SMA is an inheritable disorder that causes weakness of the skeletal muscles and wasting. It gets worse as we advance.

SMA is a result of the gene; typically, SMA1 is absent or does not function correctly. Zolgensma is a functional or working copy of the human SMN gene.Zolgensma is an all-in-one treatment that is administered through an intravenous (IV) infusion. This treatment was approved by the US Food and Drug Administration (FDA) in 2019.

How to take Zolgensma?

Zolgensma is a prescribed gene therapy that is used for children younger than two years old suffering from the condition known as spinal muscular atrophy (SMA). Zolgensma is administered as an infusion once into a vein. Zolgensma is not being evaluated in patients suffering from advanced SMA.

  • Zolgensma is administered via an IV infusion for 60 minutes.
  • Zolgensma is administered along with corticosteroid therapy. The treatment begins the day before the infusion of Zolgensma. It is extended for a total of 30 days.
  • Inform your child's doctor right away in the event that your child's skin or eyes' whites appear yellow, especially if they fail to take an injection of corticosteroid or vomit it out, or if they experience an increase in alertness.
  • Prior to and following the time your child gets Zolgensma, keep an eye out for:
    • Infections occurring prior to or after a zolgensma can result in worse complications. Consult your doctor right away whenever you observe any signs of an infection that could be a problem, like wheezing, coughing, or sneezing. You may also experience a running nose, sore throat, or fever.
    • The decrease in platelet count could happen following an injection of Zolgensma. Get medical attention immediately when the patient notices sudden bleeding or bruises.
    • Thrombotic microangiopathy (TMA) is reported to develop around one week following Zolgensma infusion. The caregiver should seek medical attention when a patient shows any symptoms or signs of TMA that are not expected, like bleeding, bruising, seizures, or a decrease in urine output.

Do I have to take steps to protect the waste products of the patient's body?

In the short term, small amounts of Zolgensma could be present in the stool of the patient. Be sure to wash your hands well whenever you come into contact with bodily waste for a period of one month following the infusion of Zolgensma. Disposable diapers must be placed in plastic trash bags that are disposable and thrown away with the rest of your garbage.

Important information

  • Zolgensma may increase the levels of liver enzymes and can cause severe liver damage and acute liver failure.
  • Your child will be given an oral corticosteroid prior to and after an infusion of Zolgensma. They will also undergo regular blood tests to check the liver's function.
  • Inform your child's doctor right away when the skin or the eyes' whites are yellowish, in the event that they fail to take a dose of corticosteroid, if they vomit it out, or if they notice a decline in alertness.

Before you take this drug

Before you give your child Zolgensma, Talk to your child's doctor about

  • Vaccinations to determine if modifications to the schedule of vaccinations are necessary to allow treatment with corticosteroids.
  • Protection against respiratory syncytial viruses (RSV) is highly recommended.

Details on dosage

The dosage for Zolgensma is 1.1 1014 vector genomes (vg) per kilogram of body mass.

Side effects of Zolgensma?

The most frequent adverse effects seen in patients receiving Zolgensma included high liver enzymes and vomiting.

This is not the only possible side effect associated with Zolgensma.

Contact your child's physician to get medical advice on the side effects that are bothersome or don't go away completely.

You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or, or Novartis Gene Therapies, Inc. At 833-828-3947.