Discover groundbreaking treatments for spinal muscular atrophy. These treatments offer hope and improved quality of life for patients and their families. If you or a loved one has spinal muscular atrophy, empower yourself with knowledge.
What Is Spinal Muscular Atrophy?
Spinal muscular atrophy (SMA) is a rare genetic disorder that affects the nerve cells (neurons) in the spinal cord and brain.1 These neurons control muscle movement, and when they are damaged or lost, it leads to progressive muscle weakness and atrophy.
Spinal Muscular Atrophy Treatment
There is currently no cure for SMA, but there are several treatments available that can help slow the progression of the disease and improve the quality of life for those affected.2 These treatments include:
- Nusinersen (Spinraza): Nusinersen is a medication that is injected into the spinal canal. It works by increasing the production of a protein called survival motor neuron (SMN), which is essential for the function of motor neurons. Nusinersen has been shown to be effective in improving motor function and survival in infants and children with SMA.
- Risdiplam (Evrysdi): Risdiplam is a medication that is taken orally. It works by increasing the production of SMN protein in the same way as nusinersen. Risdiplam has been shown to be effective in improving motor function and survival in infants, children, and adults with SMA.
- Onasemnogene abeparvovec (Zolgensma): Onasemnogene abeparvovec is a gene therapy that is administered as a one-time infusion. It works by delivering a functional copy of the SMN gene to motor neurons. Onasemnogene abeparvovec has been shown to be effective in improving motor function and survival in infants with SMA.
Discover Treatment Options for Spinal Muscular Atrophy
In addition to the three main treatments listed above, there are a number of other treatment options that may be beneficial for people with SMA. These include:
- Physical therapy: Physical therapy can help to improve muscle strength and flexibility, and it can also help to prevent contractures (permanent shortening of the muscles).
- Occupational therapy: Occupational therapy can help people with SMA to learn how to perform everyday activities, such as eating, dressing, and bathing.
- Speech therapy: Speech therapy can help people with SMA to improve their speech and communication skills.
- Respiratory therapy: Respiratory therapy can help people with SMA to manage their breathing problems.
- Nutritional support: Nutritional support can help people with SMA to maintain a healthy weight and to get the nutrients they need.
Empowering Patients With SMA Treatment Advancements
The development of new treatments for SMA has led to a significant improvement in the outlook for people with this condition. With early diagnosis and treatment, many people with SMA are now able to live full and active lives. However, there is still much work to be done, and researchers are continuing to develop new and more effective treatments for SMA.
If you or someone you know has SMA, it is important to talk to a doctor about the available treatment options. Early diagnosis and treatment can make a big difference in the outcome of the disease.
Learn More About Spinal Muscular Atrophy Treatment
With innovative therapies, patients have access to treatments that can significantly slow the disease’s progression and enhance their quality of life. These treatments, combined with supportive therapies like physical, occupational, and respiratory therapy, underscore the importance of an integrated approach to managing SMA. Empowering patients and families with knowledge about these options is crucial for early diagnosis and intervention, which are key to optimizing outcomes in SMA.