It costs 18 rupees for all of Zolgensma, the most expensive drug in the world

Do you know which is the most expensive drug in the world, and which illness is it used to cure? The name of the drug is Zorgensma manufactured by the Swiss multinational pharmaceutical company Novartis.

The virus has infected the life-changing drug Zolgensuma, considered the world’s most expensive drug and approved by the United Kingdom National Health Service (NHS) to treat rare hereditary diseases.

The reported drug with a list price of 18 chlores per dose was first approved for use in the United States and more recently in the United Kingdom.

This one-off gene therapy is used to treat certain severely ill infants Spinal muscular atrophy.. This condition causes muscle weakness, which affects movement and breathing.

According to the website of the Rare Diseases Association of India, there are more than 300,000 children with spinal muscular atrophy (SMA), but with little awareness and support.

Zolgensuma was first approved by the US Food and Drug Administration on May 24, 2019.

This made it the first gene therapy approved to treat children under 2 years of age, the most severe form of spinal muscular atrophy (SMA) and a major genetic cause of infant mortality. ..

The virus used in Zorgensma gene therapy functions as soon as it is introduced into the patient, and the patient’s body begins to produce antibodies.

If they give a second injection of the virus, it does not work because they develop immunity to it and the antibodies fight it. This means that only one injection of treatment can be used effectively.

What is Spinal Muscular Atrophy?

SMA is a rare hereditary disease caused by mutations in the survival motor neuron 1 (SMN1) gene. This gene encodes the survival motor neuron (SMN) protein. It is a systemic protein that is important for the maintenance and function of specialized nerve cells called motor neurons.

Motor neurons in the brain and spinal cord control the movement of muscles throughout the body. In the absence of sufficient functional SMN protein, motor neurons die, weaken, and often lead to fatal weakness.

SMAs caused by mutations in the SMN1 gene are generally divided into several subtypes based on age and severity of onset. Infant-onset SMA is the most severe and most common subtype.

Children in this condition have problems raising their heads, swallowing, and breathing. These symptoms may appear at birth or by the age of 6 months.

How does Zolgensuma work?

Zolgensma is an adeno-associated virus vector-based gene therapy that targets the causes of SMA.

This vector delivers a fully functional copy of the human SMN gene to target motor neuron cells.

Zolgensma uses a harmless genetically engineered virus to increase SMN protein levels.

When injected into the blood (by “intravenous injection”), the virus can travel throughout the body.

It reaches a variety of different cells to help recover some of the SMN proteins that are lost due to the condition.

Zorgensma is indicated for the treatment of children under 2 years with SMA.

This product is a single intravenous dose that results in the expression of the SMN protein in the child’s motor neurons.

Zolgensma is an SMN-enhancing therapy that works by replacing the missing or mutated SMN1 gene through a virus named AAV9.

This virus carries the replacement gene into the body. The virus “infects” cells with new DNA.

This improves muscle movement and function, and the survival rate of children with SMA.

The dosage is determined based on the patient’s weight.

Side effects of Zolgensuma

The most common side effects of Zolgensuma are elevated liver enzymes and vomiting. Zolgensma has a framed warning that serious acute liver damage may occur.

Patients with existing liver dysfunction may be at increased risk of experiencing severe liver damage.