FAQs

The following are some of the most frequently asked questions associated with SMA.

What is SMA?

SMA (spinal muscular atrophy) is a disease that robs people of physical strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat, or breathe. It is the number one genetic cause of death for infants. 

Learn more about SMA.

What are the different types of SMA?

There are four primary types of SMA: type I, II, III, and IV. The type of SMA is based on age of onset and the highest physical milestones achieved.

Learn more about the types of SMA.

What are the symptoms of SMA?

Often, the first sign of SMA type I is loss of muscle tone in very young infants. This is called hypotonia or “floppy baby syndrome.” In SMA type II and type III, an older infant or toddler may be delayed in meeting physical milestones, or fail to meet these milestones entirely.

Learn more about the symptoms of SMA.

How is SMA diagnosed?

SMA is diagnosed through genetic testing. Genetic testing, done via a simple blood test, is necessary because many conditions have symptoms similar to SMA. 

Learn more about how SMA is diagnosed.

My child was just diagnosed with SMA. What happens now?

We know that an SMA diagnosis can be overwhelming. You may be confused, scared, frustrated, or uncertain—and your emotions may change from day to day, sometimes even hour to hour. But you are not alone. You are now part of Cure SMA: a network of families, researchers, clinicians, and other professionals who are determined to make a difference.

Learn more about newly diagnosed care packages and the other resources we offer for families dealing with a recent SMA diagnosis .

What causes SMA? Is SMA genetic? 

SMA is a recessive genetic disorder. This means that both parents must pass the faulty gene on for the child to have SMA. If two carriers have a child, that child has a 25% chance of having SMA.

Learn more about the genetics of SMA.

What if I’m a genetic carrier of SMA?

Approximately 1 in 50 people is a genetic carrier for SMA. Carriers don’t have SMA, but they can pass the mutated gene on to their children. Genetic testing is available to determine carrier status, and carriers have a number of reproductive options, including prenatal testing and PGD.

Learn more about being a carrier.

Is there a treatment or cure for SMA?

Currently, Spinraza is the only FDA-approved treatment for SMA. In addition, there are currently 17 drug programs in development, five of which are being tested in clinical trials. But our work is not done. We know what we need to do to develop and deliver effective therapies. And we're on the verge of further breakthroughs that will continue to change the course of SMA for everyone affected—from infants to adults—and eventually lead to a cure. 

Learn more about Cure SMA’s approach to research and how we’re working toward a cure. 

What is happening in SMA research?

Today, we have 18 drug programs in development, including seven now in clinical trials. This means we’re getting closer and closer to an FDA-approved therapy for people affected by SMA. Of these ongoing drug programs, half have been funded by Cure SMA.

Learn more about the latest research developments and how our drug pipeline is taking shape.

Are there any clinical trials or studies that I can participate in?

There are a number of clinical trials in various stages of progress. Some are currently recruiting participants, and each trial has its own guidelines on who can participate.

Learn more about participating in a clinical trial and how to locate open trials near you.

What resources does Cure SMA offer for families affected by SMA?

Our family support department is committed to offering compassionate, respectful, unbiased support to all members of the SMA community. This includes care packages for those who are newly diagnosed, information packets covering many of the issues important to families affected by SMA, the Annual SMA Conference, grief and loss support, information on daily life with SMA, an equipment loan pool, and more.

Learn more about the support and care resources we offer.

What about breathing or other SMA medical issues?

Cure SMA provides information about a variety of medical issues associated with SMA, including palliative care, breathing, nutrition, musculoskeletal difficulties, and medical equipment. This information is beneficial both for families and medical providers.

Learn more about medical issues, including our care series booklets.

What is palliative care?

Palliative means relieving or soothing symptoms without curing the underlying disease. Though palliative care is usually associated with end-of-life decisions, it is broader than that. Palliative care aims to provide comfort for children and families, and to uphold quality of life: physically, psychologically, socially, emotionally, and spiritually. 

Learn more about palliative care.

How can I meet other families affected by SMA?

Cure SMA has 34 volunteer-led chapters across the U.S. These chapters provide a base of community and support for families affected by SMA, whether sharing practical advice or simply having someone nearby who understands what you are facing. Many chapters also organize fundraising events, get-togethers, or participate in local advocacy and awareness.

Cure SMA also hosts the Annual SMA Conference. Our annual conference is an invaluable resource for the entire SMA community, bringing together researchers, healthcare professionals, and families to network, learn, and collaborate.

Learn more about our community and local support.

How can I donate to Cure SMA?

Cure SMA accepts donations online, over the phone, or via mail. 

We’ve already invested over $59 million in research, and your support will allow us to continue funding the discoveries that will lead to a treatment and cure for SMA. Your gift will also support vital programs for families affected by SMA. Cure SMA is a 501(c)(3). All contributions are tax-deductible.

Make a donation today.

How can I plan an event to support Cure SMA?

Our work would not be possible without all the dedicated supporters who raise money and awareness for Cure SMA. Each year, our families and friends organize and participate in fundraisers—including more than 300 events throughout the country—that bring the entire SMA community closer to a treatment and cure. Our development team will provide the logistical and fundraising support you need to make your event successful and meaningful.

Get started by submitting a fundraising interest form.

What events are happening in my area?

Our event calendar makes it easy to find an event happening near you—whether you are looking for a Walk-n-Roll, gala or dinner event, golf tournament, marathon team, or another unique and exciting event.

Search our calendar by location, event type, and more.

What is the Annual SMA Conference?

Our Annual SMA Conference is really three separate events—one for families, one for researchers, and one for healthcare professionals—that run in parallel, so that all three groups can interact with and learn from one another. The conference is an invaluable resource for the entire SMA community, bringing together researchers, healthcare professionals, and families to network, learn, and collaborate. Traditionally held in June, the conference location changes from year-to-year.

Learn more about location, schedule, and registration deadlines for the Annual SMA Conference.

I’m an SMA researcher—how can I get involved?

Cure SMA has invested over $59 million in SMA research. We currently offer funding for research projects in three areas: basic research, drug discovery, and clinical care. We also host the SMA Research Group meeting. The world’s largest conference devoted to SMA research, this yearly event is held in tandem with our family conference and CME conference. This allows researchers, healthcare providers, and families to interact with and learn from each other in person. When appropriate, Cure SMA also uses its connections to the SMA community to help researchers recruit for clinical trials.

Learn more about the resources available to you as a researcher.

I’m a doctor or nurse or other healthcare provider looking for more information—where should I start?

Cure SMA’s commitment to the entire SMA community extends to those charged with providing care—including physicians, specialists, nurse or nurse practitioners, genetic counselors, physical or occupational therapists, nutritionists, social workers, and more. We educate health care professionals and the public about SMA, to enhance the quality of care and strengthen the support available to families. We work directly with clinicians, medical doctors, specialists, and skilled caregivers to ensure that patients have access to the best possible care. Our Continuing Medical Education conference provides a comprehensive curriculum for primary care providers about the diagnosis and care of SMA.

Learn more about the resources available for healthcare providers.

Have a question that isn't covered here?

Email us at info@curesma.org or call 800.886.1762 and we'll be glad to help answer it.

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