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What are the best treatments for Spinal Muscular Atrophy?

See the best treatments for Spinal Muscular Atrophy here

Spinal Muscular Atrophy treatments

Best Treatments for Spinal Muscular Atrophy


Spinal Muscular Atrophy (SMA) is a genetic disorder that affects the motor neurons in the spinal cord, leading to muscle weakness and atrophy. It is a progressive condition that can significantly impact a person's quality of life. While there is currently no cure for SMA, there are several treatments available that can help manage the symptoms and improve the overall well-being of individuals with this condition.



1. Disease-Modifying Therapies


Disease-modifying therapies are a major breakthrough in the treatment of SMA. These therapies aim to modify the course of the disease by addressing the underlying genetic cause. The two most prominent disease-modifying treatments for SMA are:




  • 1.1. Spinraza (nusinersen): Spinraza is an FDA-approved medication that works by increasing the production of the survival motor neuron (SMN) protein, which is deficient in individuals with SMA. It is administered through a series of spinal injections and has shown significant benefits in improving motor function and slowing disease progression.

  • 1.2. Zolgensma (onasemnogene abeparvovec): Zolgensma is a gene therapy that delivers a functional copy of the SMN1 gene directly to motor neurons. It is a one-time intravenous infusion and has demonstrated remarkable results in infants with SMA, including improved motor skills and increased survival rates.



2. Supportive Care


Supportive care plays a crucial role in managing the symptoms and complications associated with SMA. It involves a multidisciplinary approach to address the various aspects of the condition. Some key components of supportive care include:




  • 2.1. Physical Therapy: Physical therapy is essential for individuals with SMA to maintain muscle strength, flexibility, and range of motion. It focuses on exercises and techniques that help improve motor function and prevent contractures.

  • 2.2. Occupational Therapy: Occupational therapy aims to enhance daily living skills and promote independence. It focuses on activities such as self-care, fine motor skills, and adaptive equipment to facilitate functional abilities.

  • 2.3. Respiratory Care: SMA can affect respiratory muscles, leading to breathing difficulties. Respiratory care involves monitoring lung function, providing respiratory support when necessary, and implementing techniques to prevent respiratory infections.

  • 2.4. Nutritional Support: Maintaining adequate nutrition is crucial for individuals with SMA, as muscle weakness can affect swallowing and feeding. Nutritional support may involve dietary modifications, feeding techniques, and, in some cases, the use of feeding tubes.

  • 2.5. Psychological Support: SMA can have a significant emotional impact on individuals and their families. Psychological support, including counseling and support groups, can help address the psychological and emotional challenges associated with the condition.



3. Assistive Devices


Assistive devices can greatly enhance the mobility and independence of individuals with SMA. These devices are designed to compensate for muscle weakness and facilitate daily activities. Some commonly used assistive devices include:




  • 3.1. Wheelchairs: Wheelchairs provide mobility for individuals with SMA who have difficulty walking or standing. There are various types of wheelchairs available, including manual and power wheelchairs, depending on the individual's needs and capabilities.

  • 3.2. Orthoses: Orthoses, such as braces and splints, can help support weak muscles and improve stability. They are often used to assist with walking, standing, and maintaining proper posture.

  • 3.3. Communication Devices: Individuals with severe SMA may experience difficulties with speech. Communication devices, such as speech-generating devices or eye-tracking technology, can enable effective communication.



4. Clinical Trials


Participating in clinical trials can provide access to experimental treatments and contribute to the advancement of SMA research. Clinical trials evaluate the safety and efficacy of potential therapies, including new medications and gene therapies. It is important to consult with healthcare professionals and consider the potential risks and benefits before participating in a clinical trial.



It is crucial for individuals with SMA to work closely with a specialized healthcare team, including neurologists, physiatrists, genetic counselors, and other specialists. The treatment approach should be tailored to the individual's specific needs and may evolve as new therapies and interventions become available.


Diseasemaps
8 answers
There is no treatment at this stage. Nusinersen/Spinraza is in a final phase of trials

Posted Feb 22, 2017 by Daniel 1011
Physiotheraphy

Posted Feb 23, 2017 by ugyen 1000
nusinersen/Spinraza

Posted Feb 25, 2017 by Andrea 500
There is currently no cure for SMA. However, the FDA has recently released a possible treatment called Spiranza in the states.

Posted May 2, 2017 by Crystal Rondeau 755
Спинраза. В настоящее время другое лечение отсутствует в мире

Posted Mar 20, 2019 by Светлана 300
Translated from spanish Improve translation
Through regenerative therapies and is currently Spiranza.

Posted May 10, 2017 by Carmen 1370
Translated from french Improve translation
For my type a kine maintenance type massage with regular physical activity (but at your level,if you exercise with someone who does not have this disease, it may be that he does not understand why you are so no(need to say what that is:P)so it will be important that it starts with your level, and especially not the reverse

Posted Nov 22, 2017 by 2000

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Charlotte & Wayne lost their first daughter Annabelle Rose when she was just 7 months and 12 days old due to a genetic muscle wasting condition called Spinal Muscular Atrophy. Annabelle had Type 1 which is the most severe form of the condition and ta...
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My son Grady was born on May 15th 2015. He wasn't due until June 19th but since I had polyhydraminos my water broke on May 13th. I had noticed while I was pregnant that Grady didn't move near as often or as much as my daughter did during my first pre...
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I was born on March 5, 1949, in a poor neighborhood of Lima, Peru, in a time where most houses had no electricity and radio music was a luxury only afforded by the few. The television had not yet come to the country and Internet was unimaginable. htt...
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My daughter has a SMA type1 , the start of the disease in 5.5 months. She is 17 months. She is breathing on her own. Little holding her head. Raises handle. She begins to talk.
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No less than two neurologists have diagnosed me with SMA, but I have to say, I'm pretty sure I DON'T have it. I'm strong, graceful and powerful... the only symptoms I'm experiencing are speech slurring, weak tongue and lips and mild difficulty breat...

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