Is spinal muscular Atrophy curable?

It’s not currently possible to cure spinal muscular atrophy (SMA), but research is ongoing to find new treatments. Treatment and support is available to manage the symptoms and help people with the condition have the best possible quality of life.

Can SMA type 1 be cured?

There’s currently no cure for SMA, but the Food and Drug Administration (FDA) has recently approved several new treatments for both type 1 and type 2 SMA, including innovative gene therapies, with many more potential treatments on the horizon.

Is spinal muscular Atrophy reversible?

There’s no cure, but treatments can improve some symptoms and, in some cases, help your child live longer. Researchers are working to find new ways to fight the disease. Keep in mind that every child or adult who has SMA will have a different experience.

Can you survive with SMA?

Children with type 2 or type 3 SMA may live full lives depending on the severity of symptoms. People who develop SMA during adulthood (type 4) often remain active and enjoy a normal life expectancy.

How SMA is caused?

Most kinds of SMA are caused by a problem with a gene called the SMN1 gene. The gene does not make enough of a protein needed for the motor neurons to work normally. The motor neurons break down and can’t send signals to the muscles. A child with SMA gets one copy of the SMN1 gene from each parent.

Is SMA painful?

Overall, pain in this population of SMA patients appears to be comparable to that of people with osteoarthritis or chronic low back pain. Despite SMA patients being generally protected from severe pain, younger SMA patients do experience pain at heightened rates.

Can motor nerves repair themselves?

When one of your nerves is cut or damaged, it will try to repair itself. The nerve fibres (axons) shrink back and ‘rest’ for about a month; then they begin to grow again. Axons will regenerate about 1mm per day.

Can spinal nerves regenerate?

Adult nerve cells in the spinal cord don’t regrow after damage. Why they don’t, and how they might be encouraged to do so, have been areas of extensive research. Axons require a great deal of energy to regrow.

How does spinal muscular atrophy (SMA) affect the body?

Due to a mutation in the survival motor neuron gene 1 ( SMN1 ), individuals with spinal muscular atrophy (SMA) don’t produce survival motor neuron (SMN) protein at high enough levels. Without this protein, the motor neuron cells shrink and eventually die. This causes debilitating and potentially fatal muscle weakness.

What is SMA disease?

SMA is a progressive neurodegenerative disease that affects the motor nerve cells in the spinal cord and impacts the muscles used for activities such as breathing, eating, crawling, and walking. It is the number one genetic cause of death for infants. About Spinal Muscular Atrophy

What are the treatment options for spinal muscular atrophy?

Many people with SMA benefit from physical and occupational therapy and assistive devices, such as orthopaedic braces, crutches, walkers and wheelchairs. These treatments may also help: Disease-modifying therapy: These drugs stimulate production of SMN protein. Nusinersen (Spinraza®) is for children ages 2 to 12.

What are the treatment options for SMA?

One way of treating SMA is to increase the amount of survival motor neuron protein in the body. These ways of treating SMA are often called “SMN-based” or “SMN-enhancing” approaches.

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