SMA Stem Cell Treatment In India

In May 2019, the FDA approved onasemnogene abeparovec- xioi gene therapy for children less than 2 years old who have infantile-onset SMA. A safe virus delivers a fully functional human SMN gene to the targeted motor neurons, which in turn improves muscle movement and function and also improves the chances of survival. In august 2020, the FDA approved the orally administered drug risdipalm to treat patients age two months of age or older with SMA.

Physical therapy, occupational therapy and rehabilitation may help improve posture and prevent joint mobility and slow muscle weakness and atrophy. Stretching and strengthening exercises may help in reduce spasticity, increase range of motion and keeps circulation flowing. Some individuals require additional therapy for speech, chewing and swallowing difficulties.

Heat therapy and heat pressure may relieve muscle pain. Assistive devices such as supports or braces, orthotics, speech synthesizers and wheelchairs might help some people retain independence. Proper nutrition and a balanced diet might help in preserving muscle mass and strength. Regular blood tests would help in understanding the progressing deficiencies better and aid in designing the diet accordingly.

During such periods it becomes essential to maintain the weight of the patient and preserve strength and noninvasive ventilation due to muscle weakness in the neck, throat or chest during day time.

Disease-modifying Therapy

These drugs stimulate the production of SMN protein. Nusinersen is for children ages 2 to 12. Your provider injects the drug into the space around the spinal canal. A different medication, risdaplam, is a medication orally taken to put a stop upon the symptoms.

Genre Replacement Therapy

Children younger than two may benefit from a one–time intravenous infusion of a drug called onasemnogene abeparvovoec-xioi. This therapy replaces a missing or faulty SMN1 gene with a functioning gene.

Since Stem Cell therapy cannot possibly bring and fervent changes into the health of the patients suffering from SMA, stem cell service providers such as stem cell care India can still help you in betterment. Their alternative therapies, as well as supportive therapies, can help in bringing stability in the life of the patient. Plus, depending upon the patient’s conditions, the care provider can also advise specific lifestyle changes which would help improve the fast-deteriorating conditions. Plus, in such conditions, preserving muscle tomes in limbs is a big challenge, and physiotherapists and occupational therapists can help in such endeavors.

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Is Stem Therapy Treatment For SMA Permanent

The underlying cause behind SMA is a missing or severely mutated gene known as SMN1, which produces a protein called SMA. This protein is needed for humans to have healthy motor neurons that act as connections for the spinal cord to signal muscles throughout the body. Without the SMN protein, our nerve cells begin the process of atrophy and would eventually die, causing the symptoms of weak, limp muscles. To cure or bring betterment into the disease and its progression, a lot of research have been brought to surmise; according to findings, here are a few of the ways through which changes can be brought into the disease.

Stem Cell Therapy

Are introduced to replace the affected motor units, but their application for this disease is yet to be explored. Various types of pluripotent stem cells are being tested, including the one produced from the patient’s fibroblasts which could ensure immunologic and gene compatibility with the patient’s condition. Stem Cell Care India is one of the most prime institutions providing Stem Cell Therapy

Small Molecular Therapy

The therapy consists of increasing the SMN2 expression through medication or gene therapy. It increases the amount of full-length SMN mRNA and, therefore, the amount of SMN protein produced in the patient’s body. Molecules that have been tested with little success include histone deacetylase inhibitors, aminoglycosides and quinazoline compounds, which, while under research, increased full-length SMN transcription in mouse models but not in human clinical trials.

RNA-Based therapy

Antisense oligonucleotide ( ASO therapy is used to increase the rate of inclusion of exon 7 into the mRNA transcripts of SMN2. These are RNA sequences which bind the complementary sequences in an intron or exon of interest splicing silencer N1 in SMN2, an ASO which blocks the intronic repressor element1, and one which targets the junction of intron 7 and exon 8 but also increases the activity of hnRNP-A1 preventing exon8 inclusion whilst increasing exon7 inclusion..

Viral-medicated gene replacement therapy

Under this therapy use of vectors was demonstrated by the adenovirus-associated viral vector serotype9 in baby mice in research which increased the SMN1 expression by 60% and increased their life span by a period of about 400 days. Time is of the essence in these therapies as an early institution of such treatment is essential for normalizing the health of the motor neurons before the critical point when they are doomed to undergo apoptosis.

Supportive care is essential in prolonging the lifespan of SMA patients, especially those with severe motor neuron loss. Such patients would suffer from problems such as

  • Respiratory distress and aspiration pneumonia
  • Nutritional deficiencies due to feeding difficulties because of weak sucking, swallowing and chewing actions.
  • Progression of the disease can also lead to secondary kyphoscoliosis and joint contractures because of weak antigravity muscles.
  • Rehabilitation because of inability to stand or walk independently in all but the mildest cases
  • Emotional distress and social problems.
Respiratory care

Respiratory care is one of the most important parts of management for patients suffering from SMA. With the progression of the disease, the muscles involved with breathing break down and lead to difficulties in breathing. Patients should be provided help to maintain muscle tone in the torso to help in breathing effectively.

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SMA Treatment with Stem Cell Therapy In India

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.

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