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Tips for Staying on Spinal Muscular Atrophy (SMA) Therapies

Updated on September 15, 2022
Medically reviewed by
Evelyn O. Berman, M.D.
Article written by
Kelly Crumrin

  • Costs for spinal muscular atrophy (SMA) medications are high, but financial assistance programs can connect you with resources to help.
  • Travel tips can help make transportation to SMA treatment centers less challenging.
  • It can be hard to stick with supportive therapies, but they are vital to help people with SMA feel their best.
  • The advent of new SMA treatments and newborn screening is expected to revolutionize many aspects of SMA care.

Spinal muscular atrophy (SMA) is a neuromuscular disorder that causes progressive muscle weakness and loss of motor function. SMA can affect young children, adolescents, adults, and their loved ones. People with SMA often face obstacles to starting — and sticking with — effective treatment. Understanding all of your options and resources is an important first step in the treatment decision-making process.

Cost of SMA Treatments

The three medications approved by the U.S. Food and Drug Administration (FDA) for spinal muscular atrophy are among the most expensive drugs in the world. Even excluding the cost of these drugs, SMA is already a very expensive rare disease — annual health care costs for someone with SMA type 1 are estimated at $137,627 per year. However, there are programs in place that can help more people access potentially lifesaving SMA treatment options.

Financial Resources for SMA Treatment Costs

  • If you have commercial insurance (not provided through a government program), you may be eligible for a $0 copay through Biogen’s SMA360 Program for nusinersen (Spinraza).
  • Avexis, the maker of onasemnogene abeparvovec-xioi (Zolgensma), has created the OneGene Program to help family members navigate insurance approval and financial assistance resources for children with SMA.
  • Genentech’s MySMA Support service provides case managers who can help with insurance support and assistance finding financial options for risdiplam (Evrysdi).
  • For those participating in clinical trials, the cost of treatment may be covered by the trial’s sponsor.

It is worth noting that, although a health insurance program may cover one of the medications approved for SMA, some insurance carriers may place limitations on coverage such as proof of symptoms or disease progression, specific genetic testing results, or age limits other than those specified by the FDA label.

In April 2019, the influential Institute for Clinical and Economic Review (ICER) issued a report recommending that manufacturers lower the prices of Spinraza and Zolgensma.

Traveling for SMA Treatment

According to one study of 1,476 Americans with SMA, the median distance to the nearest SMA treatment center was just over 54 miles. People with SMA living in rural areas had to travel much farther — a median distance of 200.1 miles. Over 70 percent of those who participated in the study reported a high burden of travel to access their nearest treatment center. The cost of travel can also be a factor, especially for those who require air travel and hotel accommodations.

Finding Treatment Centers

Cure SMA’s Find a Location mapping tool makes it easy to find the nearest clinic offering treatment for SMA for children or adults. ClinicalTrials.gov offers a simple search tool to locate clinical studies for SMA near you.

Tips To Make Traveling With SMA Easier

Travel can be challenging with SMA due to mobility issues and the need to transport medical equipment, such as BiPAP systems. Here are a few tips that can help make travel with SMA easier:

General Travel Tips

  • Locate another SMA family near the treatment location. They may be able to help if you experience problems with equipment. On mySMAteam, you can meet others with needs similar to yours or your child’s.
  • Carry a selection of replacement parts and tools you may need if a powered wheelchair or respiratory support device requires repair.
  • Cure SMA offers an equipment pool with free loans of items such as manual wheelchairs and car beds, which allow babies with SMA type 1 to safely travel in cars.

Air Travel Tips

  • When you make flight reservations, notify the airline about any special accommodations you or your child require.
  • If your baggage is overweight due to medical necessities, such as a powered wheelchair charger, ask for a waiver for any fees.
  • Contact the airline and the Transportation Security Administration (TSA) at least 48 hours in advance of your flight to finalize arrangements for any special accommodations you need.
  • When leaving a powered wheelchair to embark on the plane, consider wrapping it in padded packing material, attaching instructions for airline staff, or simply taking a photo to benchmark its state and settings. When the wheelchair is returned upon landing, compare it to the photo to quickly identify any changes or damage.

Treatment Adherence

Many supportive therapies can help people with SMA stay their healthiest and feel their best, but most of them require sticking to a routine, often on a daily basis. Adherence to treatment is defined as following a doctor-recommended course of medications, diet, exercise, or other treatments just as prescribed. However, adherence to supportive therapy can prove difficult for people with SMA due to symptoms like fatigue, sleep disturbances, and pain in the back and chest. In adolescents and adults with later-onset SMA, disengagement from health care is common and can further impact adherence.

For people with SMA, adherence to treatment might include:

  • Daily practice of breath stacking for those who are losing lung capacity.
  • Using BiPAP at night and a cough-assist device at least twice a day to prevent low oxygenation and respiratory infections.
  • Sticking to a meal plan to ensure adequate nutrition and manage weight.
  • Regularly performing physical therapy exercises to maintain function and range of motion.
  • Maintaining good posture every day to prevent worsening of scoliosis.

Despite evidence that these therapeutic routines are effective, many people with SMA have trouble sticking to them. With the advent of SMA medications that can potentially extend life, adherence to rehabilitative therapies may become more important than ever to restore function, maintain abilities, and improve quality of life.

Tips to Improve Treatment Adherence

In chronic conditions in general, 80 percent of people drop out of chronic care management plans prescribed by their doctors. Here are four tips to improve treatment adherence:

Stay Organized

Set up a system of reminders to keep you organized. If you’re comfortable with technology, there are smartphone apps designed to help people manage treatment regimens. You can also take a low-tech approach using sticky notes and tying therapy practices to daily activities, such as meals. For instance, you could complete physical therapy exercises before lunch each day.

Schedule Out Appointments

Scheduling out regular events that happen a few times a year, such as checkups or Spinraza administrations, can help prevent you or your child from missing an important appointment. It may be helpful to keep your calendar scheduled six or more months in advance with two-week reminders as appointments approach — perhaps more notice if a long journey is required.

Manage Discomfort

The ultimate goal of therapy is to maintain function and keep you feeling your best, but it can be hard to stick with therapy if it feels uncomfortable when you’re doing it. If you find it difficult to stick with physical therapy due to pain, or you avoid using your BiPAP due to dry mouth or sinus congestion, ask your doctor for ways to manage the discomfort.

Engage With Community

Connect with others who have SMA, either in a face-to-face support group or in online communities like mySMAteam. Set your goals together for therapy and hold each other accountable for following through. Support each other when it’s difficult and cheer each other on when goals are met.

Fewer Obstacles Ahead?

In recent years, researchers have begun to focus on ways to improve quality of life and mental health for people with spinal muscular atrophy. A 2019 study from the journal Disability and Rehabilitation emphasizes the importance of an “autonomy supportive” approach to health care for people with SMA. Ideally, each person with SMA will have access to a patient-centered, multidisciplinary team of health care professionals who support them in setting and achieving realistic goals and overcoming barriers.

According to the Institute for Clinical and Economic Review, the approval of Spinraza is expected to improve access to SMA treatment in many ways. Spinraza provided motivation for the U.S. Health and Resources and Services Administration (HRSA) to add spinal muscular atrophy to the Recommended Uniform Screening Panel for newborn screening. With early identification of babies with SMA, awareness and understanding of the condition will become more widespread among health care professionals. ICER predicts a revolutionary improvement in the infrastructure of care for SMA.

Learn more about ways to take care of yourself with SMA in these related articles:

References
  1. Droege, M., Sproule, D., Arjunji, R., Gauthier-Loiselle, M., Cloutier, M., & Dabbous, O. (2019). Economic burden of spinal muscular atrophy in the United States: a contemporary assessment. Journal of Medical Economics, 23(1), 70–79. doi: 10.1080/13696998.2019.1646263. Accessed at https://www.tandfonline.com/doi/full/10.1080/13696998.2019.1646263
  2. Travel Burden of Spinal Muscular Atrophy Patients in the United States (P1.6-070). Joseph Goble, Dong Dai, Xue Song, Nianwen Shi, Fernanda Boulos, Lu Zhang, Kristen Johnson. Neurology Apr 2019, 92 (15 Supplement) P1.6-070. Accessed at https://n.neurology.org/content/92/15_Supplement/P1.6-070
  3. Bose, M., Parab, S., Patil, S., Pandey, N., Pednekar, G., & Saini, S. (2019). Exploring spinal muscular atrophy and its impact on functional status: Indian scenario. Indian Journal of Public Health, 63(3), 254. doi: 10.4103/ijph.ijph_239_18. Accessed at http://www.ijph.in/article.asp?issn=0019-557X;year=2019;volume=63;issue=3;spage=254;epage=257;aulast=Bose
  4. Wan, H.W.Y., Carey, K.A., D’Silva, A. et al. “Getting ready for the adult world”: how adults with spinal muscular atrophy perceive and experience healthcare, transition and well-being. Orphanet J Rare Dis 14, 74 (2019). Accessed at https://doi.org/10.1186/s13023-019-1052-2
  5. Kerr, L. M., Kerr, Butterfield, R., Candee, M., … Swoboda, K. (n.d.). Spinal Muscular Atrophy. Retrieved February 2020, from https://www.medicalhomeportal.org/diagnoses-and-conditions/spinal-muscular-atrophy
  6. Lemoine, T. J., Swoboda, K. J., Bratton, S. L., Holubkov, R., Mundorff, M., & Srivastava, R. (2012). Spinal muscular atrophy type 1. Pediatric Critical Care Medicine, 13(3). doi: 10.1097/pcc.0b013e3182388ad1. Accessed at https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4334579/pdf/nihms569816.pdf
  7. DiVito, D., & Konek, S. (2010). Spinal Muscular Atrophy — Summary for Nutritional Care. ICAN: Infant, Child, & Adolescent Nutrition, 2(6), 348–354. doi: 10.1177/1941406410390938. Accessed at https://journals.sagepub.com/doi/pdf/10.1177/1941406410390938
  8. Foead AI, Yeo WW, Vishnumukkala T, Larvin M. Rehabilitation in spinal muscular atrophy. J Int Soc Phys Rehabil Med 2019;2:62-70. Accessed at http://www.jisprm.org/article.asp?issn=2349-7904;year=2019;volume=2;issue=1;spage=62;epage=70;aulast=Foead
  9. PatientEngagementHIT. (2017, August 22). 5 Ways to Improve Medication Adherence in Chronic Care Patients. Retrieved February 2020, from https://patientengagementhit.com/news/5-ways-to-improve-medication-adherence-in-chronic-care-patients
  10. Maarten J. Fischer, Fay-Lynn Asselman, Esther T. Kruitwagen-van Reenen, Marjolein Verhoef, Renske I. Wadman, Johanna M. A. Visser-Meily, W. Ludo van der Pol & Carin D. Schröder (2019) Psychological well-being in adults with spinal muscular atrophy: the contribution of participation and psychological needs, Disability and Rehabilitation, DOI: 10.1080/09638288.2018.1555864. Accessed at https://www.tandfonline.com/doi/full/10.1080/09638288.2018.1555864
All updates must be accompanied by text or a picture.
Evelyn O. Berman, M.D. is a neurology and pediatric specialist and treats disorders of the brain in children. Review provided by VeriMed Healthcare Network. Learn more about her here.
Kelly Crumrin is a senior editor at MyHealthTeam and leads the creation of content that educates and empowers people with chronic illnesses. Learn more about her here.

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