Spinal Muscular Atrophy leads to the breakdown of the nerve cells in the brain and the spinal cord, and the brain stops sending messages that control muscle movement. In the case of kids under such circumstances, kids become unable to control their heads, could not sit without help, and even walk as the disease gets worse with time; the kids might also witness trouble in swallowing and breathing. Unfortunately, no treatments have been fully able to improve the symptoms and help stop the aggressive rate of the disease progression.
Symptoms and onset of the diseases differ very differently based upon the type of SMA.
-
Type 0
The rarest and the most severe kind of SMA develops while a mother is in pregnant. Babies with this type of SMA move less in the womb and showcase symptoms of joint problems, weak muscle tone, and weak muscles for breathing. Although it is hard to bring forward, but these kids generally die due to breathing issues.
-
Type 1
In this kind of SMA. A child may be unable to support their head or sit without help. It’s possible that they might have floppy arms or legs and have problems in swallowing due to weak muscles. The biggest concern in this type of disease is that weakness presumes in the muscles that control breathing.
-
Type 2
This generally affects kids 6-18 months old. The symptoms range from moderate to severe and usually involve the legs more than the arms. Your child may be able to walk, sit and move with help.
-
Type 3
In this type of SMA, symptoms start when kids are between the age of 2-17 years old, this is the mildest form of the disease; a child suffering from this stage would be most likely to be able to stand or walk without help but may have problems running, climbing stairs or getting up from a chair after sitting. This type of SMA is also known as Jugelberg- Welander disease or Juvenile SMA.
-
Type 4
This form of SMA starts when you are an adult, it showcases symptoms such as muscle weakness,t twitching, or breathing problems. Usually, only your upper arms and legs are affected. You will have the signs throughout your life, but you can keep moving and even get better with exercises that you would practice with the help of a physical therapist.
SMA is primarily caused by mutations in the gene of the survival motor neuron. More research has been focused on developing SMN-targeted replacement therapy for SMA. Recently Stem cell transplantation has shown the potential to repair the damaged tissue and differentiate it into neurons to rescue the phenotypes of SMA in animal modes.
Other than this, there are three FDA-approved medications such as
-
Nusinersen (spinraza)
This treatment adjusts the smn2 gene and coaxes it to produce more protein. This medication is used for both children as well as adults. The medical team undertaking the treatment would inject the drug into the fluid around the spinal cord. Including preparation and recovery time, this can take at least two hours and will need to be done a few times, followed by another dose every 4 months. According to the research conducted on the subject, 405 of people who use it have showcased signs of a decrease in the progression of the disease and showcased signs of being better in health than before.
Stem Cell Therapy in such a case is still under trial, and only certain people with showcase-specific vitals can undertake it. Stem Cell Care India is one of the most prime ace institutions which can help patients in therapy. Plus, their existing support therapies can also help the patients. They design treatments according to the unique needs of the patient, and their lifestyle-altering therapies can help in improving the patient’s condition.