Launched by Roche, Evrysdi is the first and only treatment approved in India for patients with spinal muscular atrophy.

Roche’s Evrysdi is an oral treatment for adults and children over 2 months of age who suffer from spinal muscular atrophy.

Roche has begun oral treatment for adults and children suffering from spinal muscular atrophy (SMA). Named Evrysdi, this treatment is the first and only approved treatment for SMA patients in India.

What is SMA?

Spinal muscular atrophy (SMA) is a hereditary disorder that causes muscle weakness by gradually destroying motor neurons. These are nerve cells in the brain and spinal cord that allow a person to move, talk, swallow, and breathe by relaying commands from the brain to the muscles that perform these actions.

People affected by SMA have a mutation in the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of the SMN protein. This protein is essential because it controls muscle and movement and helps nerve function.

In August, we will focus on raising awareness about SMA.

SMA is usually diagnosed at about 18 months or in childhood and is the most common cause of genetic death in babies. It affects approximately 1 in 10,000 live births worldwide and 1 in 7,744 live births in India and is the leading genetic cause of infant mortality.

Currently, there is no cure for SMA. However, research is underway to develop treatments. Treatment and assistance is available to help manage symptoms. Life expectancy for people with SMA is normal.

What is Evrysdi?

Evrysdi is a powder that is mixed with a liquid and administered to individuals for 2 months or longer, either orally or via a feeding tube. It treats SMA by increasing the production of the survival motor neuron (SMN) protein.

After conducting a global clinical trial on 450 volunteers, it was approved by the Indian health authorities 11 months after being approved by the US FDA. These trials included infant and adult volunteers up to the age of 60. It also included patients who had previously been treated for SMA with other drugs.

Roche has announced that it will offer three bottles free of charge for every two bottles purchased by patients during the first two years of treatment to give people immediate access to this treatment. From the third year onwards, two bottles will be provided free of charge for each bottle purchased by the patient.

SMA type

• Known as Weldnighoffmann’s disease or infant-onset SMA. SMA type I It usually appears before 6 months of age. Without any treatment, affected children never sit or stand, and the majority of them usually die of respiratory failure before the age of two.
• And children SMA type II Symptomatology usually occurs between the ages of 6 and 18 months. They can sit without support, but they cannot stand or walk. Life expectancy is shorter, but you can live to adolescence or young adulthood.
• And children SMA type III (Kugelberg-Welander’s disease) is symptomatic after 18 months of age and can walk independently. They may initially have difficulty walking, jogging, climbing stairs, or getting up from a chair and may quiver in their hands. With proper care, they can lead a normal life.
• Individual SMA type IV You may experience mild to moderate proximal weakness, along with other symptoms, and develop the disease after age 21.

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