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Stem Cell Treatment for Spinal Muscular Atrophy

Regeneration and replacement of damaged cells

Can stem cell therapy help spinal muscular atrophy?

Spinal Muscular Atrophy (SMA) is a genetic disorder that affects the motor neurons in the spinal cord, leading to progressive muscle weakness and atrophy. SMA is caused by mutations in the survival motor neuron 1 (SMN1) gene, which is responsible for producing a protein essential for the survival and function of motor neurons. The severity of SMA can vary widely, ranging from severe forms that manifest in infancy and early childhood, leading to significant motor impairments and reduced life expectancy, to milder forms that appear later in childhood or even adulthood. Symptoms can include difficulty with motor skills, weakness in the limbs and trunk muscles, respiratory difficulties, and in severe cases, complications related to swallowing and breathing. While there is currently no cure for SMA, there are treatments available that aim to manage symptoms and slow disease progression, such as medication, physical therapy, and supportive care. Genetic testing can help diagnose SMA and guide appropriate management strategies.

The treatment for SMA depends on the type and severity of the condition. Currently, there are various treatment approaches available. For SMA caused by a deficiency of the survival motor neuron (SMN) protein, gene therapy medications like Onasemnogene Abeparvovec (Zolgensma) can be used to replace the missing or mutated SMN1 gene. Another medication called Nusinersen (Spinraza) is an antisense oligonucleotide that increases the production of SMN protein. Physical therapy and rehabilitation are also important components of treatment to help improve muscle strength, mobility, and overall function.

Stem cell therapy is another treatment option for the treatment of SMA, it has shown promise in the treatment of SMA. Over the years, patients traveled across the world and received stem cell therapy for SMA.

How does stem cell therapy treat SMA?

Stem cells possess the remarkable ability to self-renew, proliferate, and differentiate into various specialized cell types, allowing them to regenerate and repair damaged tissues. Stem cells also offer immunomodulatory effects, influencing immune responses in the body.

Stem cell therapy is an emerging and promising approach for the treatment of SMA. It involves using stem cells to replace or repair damaged cells in the affected tissues, they have the potential to differentiate into different types of cells, including motor neurons or other relevant cells types, making them a potential therapeutic option for SMA.

Mesenchymal stem cells (MSCs) have been studied in the treatment of SMA, these stem cells can be derived from various sources, including umbilical cord blood, bone marrow, or even the patient’s own cells.

In preclinical and early-stage clinical studies, stem cell transplantation has shown some promising results in improving motor function and slowing disease progression in SMA. The transplanted stem cells may help to replace lost motor neurons or provide trophic support to existing neurons.

The aim of stem cell therapy in SMA treatment is to restore or improve motor function and slow down the progression of the disease. It has great potential to alleviate symptoms, improve muscle strength, and enhance overall quality of life for individuals with SMA. Additionally, stem cell therapy aims to promote tissue repair and regeneration in affected areas of the spinal cord, contributing to long-term therapeutic benefits.

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Benefits of Stem Cell Therapy in Spinal Muscular Atrophy

Studies on stem cell therapy in SMA treatment and clinical practice indicated that stem cells can help SMA patient improve in many aspects.

How Do We Help?

Not Only Stem Cells

Stem cell therapy brings hope for many medical conditions, including spinal muscular atrophy, we believe that patients can live a better quality of life with effective and safe treatment.

We combine cutting-edge stem cell technology, the most experienced medical professionals in managing stem cell therapy, modern facilities and dedicated services that are effective to ensure the best possible treatment outcomes.

The stem cells used in our treatment are manufactured in GMP-certified laboratories, a series of processing and quality assurance procedures have been developed to meet the national and international standards, to ensure safe and effective products are delivered to our patients.

In addition to stem cell therapy, we provide comprehensive treatments that include, rehabilitation, remedies and natural therapies, each patient receives a personalized treatment plan tailored to meet his/her particular needs, in order to get the best our of the treatments.

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SMA Patient Experiences

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Spinal Muiscular Atrophy (SMA) FAQs

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder characterized by the progressive degeneration of motor neurons in the spinal cord. This leads to muscle weakness and wasting, affecting voluntary movement and basic functions such as breathing and swallowing. SMA is caused by mutations in the survival motor neuron 1 (SMN 1) gene, which results in a deficiency of the SMN protein necessary for motor neuron health and function. The severity of SMA can vary, with different types ranging from severe forms presenting early in infancy to milder forms manifesting later in childhood or adulthood. SMA is a chronic condition that requires multidisciplinary medical care to manage symptoms and maintain quality of life.

SMA is primarily caused by mutations in the survival motor neuron 1 (SMN 1) gene. This gene is responsible for producing the survival motor neuron (SMN) protein, which is crucial for the proper development and function of motor neurons in the spinal cord. Mutations in the SMN1 gene result in a deficiency or absence of functional SMN protein, leading to the progressive degeneration of motor neurons. Without healthy motor neurons, the muscles gradually weaken and waste away, resulting in the characteristic symptoms of SMA. The severity of SMA can vary depending on the amount of functional SMN protein produced by a second gene called SMN2, which acts as a backup but is less efficient in producing the necessary protein. In most cases, SMA is inherited in an autosomal recessive manner, meaning that an individual must inherit two copies of the mutated SMN1 gene, one from each parent, to develop the condition.

SMA is characterized by progressive muscle weakness and wasting due to the degeneration of motor neurons. Common symptoms include muscle weakness, particularly in the limbs, leading to difficulties with motor function such as sitting, standing, walking, swallowing, and breathing. Infants may exhibit delayed motor milestones. Respiratory problems can arise, resulting in weakened cough, shallow breathing, or difficulty clearing mucus from the lungs. Tremors or twitching may occur, and scoliosis can develop over time. The severity and progression of symptoms vary among individuals, highlighting the importance of early diagnosis and comprehensive medical care for SMA management.

SMA is diagnosed through a combination of clinical evaluation, genetic testing, and sometimes electromyography (EMG) and nerve conduction studies. Clinical evaluation involves assessing muscle strength, tone, reflexes, and motor skills, along with evaluating family history and symptoms. Genetic testing confirms the presence of mutations in the SMN1 gene responsible for SMA, typically through blood or saliva samples. EMG and nerve conduction studies may be performed to assess nerve and muscle function. Early diagnosis and genetic testing are crucial for appropriate medical care and treatment planning, as well as providing genetic counseling for families at risk of SMA.

The current treatments for SMA primarily revolve around disease-modifying therapies and supportive care. Disease-modifying therapies include gene replacement therapy (such as onasemnogene abeparvovec) and survival motor neuron 2 (SMN2) gene splicing modifiers (such as nusinersen and risdiplam), which aim to increase functional SMN protein production.

These therapies can help slow down the progression of the disease and improve motor function. Supportive care involves a multidisciplinary approach that may include physical and occupational therapy, respiratory support, nutritional management, and orthopedic interventions to optimize quality of life and manage symptoms associated with SMA. Early diagnosis and intervention are crucial for maximizing the potential benefits of these treatments.

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