The magic drug helps paralyzed children to walk

Children with spinal muscular atrophy will be very weak in the first months of life. They will have difficulty breathing, even unable to suck, swallow and often do not live for more than 2 years.

Spinal muscular atrophy (SMA) is a rare genetic disorder that often leads to premature death in pediatric patients. But a drug that has been produced and tested recently has had an amazing therapeutic effect. Even this drug allows many paralyzed children with spinal muscular atrophy to regain mobility.

This drug, called nusinersen, is attracting scientific attention because of its amazing therapeutic effect.

When the drug is used on patients with long-term illness, they gradually regain their ability to move without any assistance.

Spinal muscular atrophy can cause many serious consequences for each patient. But basically, the disease will cause nerve cells to become paralyzed and thereby weaken muscles. Within a year or two, many infected children will not be able to sit up, move their limbs or even swallow food.

This is an inherited disease that affects the part of the nervous system that controls spontaneous muscle activity. Most neurons that control muscles are located in the spinal cord. This is a muscular disease because its main effect occurs in muscles that do not receive signals from these neurons.

Children who are paralyzed due to spinal muscular atrophy will be completely treated in the future.(Image source: twitter).

"In the past, patients with this disease were almost like a dead person. But after taking the medicine, they had recovered, able to stand, sit and eat themselves again. a miracle, " said researcher Francesco Muntoni from the University of London in the UK.

Muntoni and his colleagues are among the researchers around the world who have contributed to the development of nusinersen. The drug works by inhibiting a particular gene that causes disease.

This treatment uses specially designed DNA to affect specific RNAs. RNA functions similar to a plant that produces proteins in the body. Therefore the proteins from there are also changed.

The nusinersen drug is a powerful effort by scientists to find a solution to stop this deadly spinal muscular atrophy.

While there are many positive effects, scientists still cannot be sure whether nusinersen can cure spinal muscular atrophy.

If the nusinersen line is really effective and can treat genetic defects, diseases similar to Huntington's disease, neuromuscular degeneration and Alzheimer's will also be directed. go new.

But there is still a long way to go for nusinersen therapy. Currently, the treatment must be repeated repeatedly every few months. The medicine will be injected directly into the spine.

From there, the drug will dissolve into the cerebrospinal fluid and then flow to the brain and spine. However, the side effects of the treatment process will give you a feeling of headache and back pain.