Zolgensma: Used to treat spinal muscular atrophy (SMA).

Brief information about it:

Zolgensma is a gene therapy medication used to treat spinal muscular atrophy (SMA), a genetic disorder that causes muscle weakness and wasting. It is the most expensive medication currently available.

 Uses:

Spinal Muscular Atrophy (SMA): Zolgensma is approved for the treatment of SMA in infants and young children. It is designed to replace the defective gene that causes SMA.

 Mechanism of action:

Zolgensma replaces the defective SMN1 gene with a functional copy of the gene. This allows the body to produce the protein needed for muscle function.

Elimination half life:

The elimination half-life of Zolgensma is relatively long, allowing for sustained benefits. However, the exact elimination half-life can vary depending on individual factors.

Route of Administration:

Zolgensma is administered as a one-time intravenous infusion.

 Side effects:

Zolgensma can cause side effects, including:

• Fever
• Vomiting
• Increased liver enzymes
• Infusion reactions

 Dose:

The dosage of Zolgensma is determined based on the patient’s weight. It is typically administered as a single infusion.

 Precautions:

·  Consult a doctor: Before receiving Zolgensma, consult with a healthcare professional to ensure it is safe for you and to discuss potential risks and benefits.

·  Allergic reactions: Inform your doctor if you have any allergies to medications or other substances.

·  Monitoring: After receiving Zolgensma, you will need to undergo regular monitoring to assess your response to the treatment and monitor for any side effects.