The world's most expensive injection reached Jaipur from America!

The world's most expensive injection 'Zolganesma' worth Rs 17 crore has reached Jaipur. This injection will give life to Little Heart, who is suffering from a rare genetic disease 'Spinal Muscular Atrophy'. Let us know why this special injection from America is so expensive and what is this rare disease.

Spinal muscular atrophy (SMA) is a rare neuromuscular disorder that causes weak muscles in babies, making it difficult to perform physical activities. In today's article, we will learn in detail about spinal muscular atrophy. This includes its symptoms, causes, diagnosis, and treatment possibilities.

What is Spinal Muscular Atrophy (SMA)?

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder. In this, due to a defect in an important gene, the body is not able to produce SMN1 protein in sufficient quantity. This protein is essential for nerve cells to maintain healthy muscles and their functionality. Due to a deficiency of SMN1 protein, muscles gradually become weak, due to which there is difficulty in doing physical activities.

Types of SMA

There are mainly four main types of SMA, which are divided based on the severity of the disease and time of onset of symptoms.

SMA type 1 (Werdnig-Hoffmann disease): This is the most severe type, usually appearing within a few months of birth. In this condition, babies have difficulty breathing, they are not able to even lift their heads and the development of muscles stops.

SMA Type 2 (Intermediate SMA): This is a moderately severe type, whose symptoms usually appear between 6 months and 18 months of age. Children can sit in this but have difficulty standing or walking.

SMA type 3 (Kugelberg-Welander disease): This is the least severe type, usually beginning in childhood or adulthood. In this, muscle weakness gradually increases and there is difficulty in walking.

SMA type 4 (Adult-onset SMA): This is a rare disease of SMA that occurs in adults. Its symptoms gradually increase, including fatigue, muscle weakness, and tremors.

Symptoms of SMA

Symptoms of SMA can vary depending on the type and severity of the disease, but some common symptoms include

- Difficulty in breastfeeding in infants

- Muscle weakness

- Difficulty in physical activities, such as sitting, standing Standing, or walking

- Difficulty breathing

- Muscle cramps

- Difficulty swallowing.

Causes of SMA

SMA is a genetic disease that is inherited from parents. It is caused by a defect in the SMN1 gene, which produces a protein essential for the development and survival of motor neurons. Due to bad genes, not enough protein is produced, resulting in motor neurons getting damaged and dying.

Diagnosis and treatment

of SMA SMA can be diagnosed through different methods including genetic tests, blood tests, and electromyography (EMG). Although there is no cure for SMA, some treatments can help reduce symptoms and improve quality of life. A medicine called Zolgensma has been made to treat this disease.

What is Zolgensma?

Zolgensma, developed by Swiss pharmaceutical company Novartis, is designed to treat SMA. Zolgensma delivers a functional copy of the SMN gene into motor neuron cells, improving muscle movement and function in children with SMA.

Why is Zolgensma priced so high?

According to experts, the price of Zolgensma in India is around Rs 17 crore because a lot of research and development is involved in it. Limited market size and ability to save lives are also the reasons for its high price. Although its initial cost is high, experts argue that the long-term benefits of Zolgensma may offset the healthcare costs associated with SMA treatment and care.