Welcome and thank you for reaching out to Cure SMA!

The diagnosis of spinal muscular atrophy (SMA) can be confusing, scary, frustrating, pretty much the whole gamut of emotions, but you are not alone. You are now part of Cure SMA – A network of individuals with SMA, families, researchers, clinicians, and other health professionals who are determined to make a difference.

Founded by a group of parents in 1984, Cure SMA will provide unbiased support for all those dealing with SMA and will fund the most promising research. We know that there are many parts of the world that are lacking access to treatment and care for SMA and as such, we are including many resources for you to use and share below. Some resources are those created by Cure SMA, while others are resources to support you in your advocacy efforts in your country and communities. Please do not hesitate to contact us with any questions.

Research & Treatments

The basic research that Cure SMA has funded, through research grants to institutions around the world, has delivered major discoveries:

  • We know the cause of SMA, which means we can develop treatments that correct the underlying cause of the disease rather than just reduce symptoms.
  • We identified a “back-up gene” for SMA, which means we have a straightforward drug target already in the body: a built-in switch for new therapies to work on.

Using this knowledge, we now have multiple therapies approved by the U.S. Food and Drug Administration (FDA) that target the underlying genetics of SMA, over half a dozen clinical trials testing SMA therapies, and many programs in various stages of drug development. Click here to learn more about the drug pipeline.

FDA Approved Therapies for SMA

There are currently three FDA-approved therapies for SMA: Spinraza, Zolgensma and Evrysdi.

All treatment decisions should be made in consultation with a qualified healthcare professional. Each company below has dedicated staff to help families navigate the process of pursuing treatment both locally and internationally:

Spinraza Contact: Biogen
Staff name: Family Access Manager
Phone: 1.866.633.4636
Email: [email protected]

Zolgensma Contact: Novartis
Staff name: Patient Resource Manager
Phone: 1.833.828.3947
Email: Find your Novartis Zolgensma Contact Here

Evrysdi Contact: Genentech
Staff name: Partnership & Access Liaison
Phone: 1.833.387.9734
Email: Find your Genentech Roche Evrysdi Contact Here

 

Financial Assistance for SMA Treatments

Cure SMA is unable to cover treatment costs or promote fundraising efforts for individual patients (in the US or internationally) within the scope of our organization's mission. Cure SMA provides support and information and helps to fund research on SMA. We greatly suggest that you reach out to the pharmaceutical companies listed above and nearby organizations mentioned under the Patient Advocacy Organizations and Groups on this page, as well as the SMA Support System Facebook Group page. Many families and individuals have sought financial assistance for treatment and care and are able to share their experience and advice on that platform.

Support Resources

Care Series Booklets

Click here to access and download all of our Cure SMA Care Series booklets in a variety of languages. These discuss topics from genetics of SMA, breathing basics, musculoskeletal system, and much more and most are available in up to 13 languages.

Resource Guides

Cure SMA also offers a wide range of resource guides which cover a range of topics to enrich daily living at home and in the community. Here is the link to access all currently available forms and to request a PDF copy: https://survey.alchemer.com/s3/7604215/Cure-SMA-Resource-Request-Form

Patient Advocacy Organizations and Groups

Patient advocacy organizations exist to empower individuals, in this case, in the rare disease community in advocating for treatments, care, and access in their local communities and countries. These organizations provide tools and support for your own advocacy efforts, which is vital in ensuring access is available for treatment and care in all countries.

PAG Spreadsheet: -https://www.curesma.org/International-Advocacy-Organizations

Global Genes – https://globalgenes.org/

Rare Diseases International - https://www.rarediseasesinternational.org/

Support Resources

Connect with Cure SMA on social media!

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Connect with the SMA Community in Peer Led Support Groups on Facebook

Online support groups can be a wonderful source of information and support and allow you to connect directly with others in the community from all over the world. Please note these online groups are run by members in the SMA community and Cure SMA does not run or monitor the groups in the list. The extent to which you participate in any group is entirely up to you but can be helpful in connecting with many others.

SMA Support System: https://www.facebook.com/groups/SMASupportSystem/

This is a peer-led group where thousands of SMA community members connect from all over the world and are often able to share helpful local support and advice.

Treatment Specific Peer Led Facebook Groups

Zolgensma gene therapy for SMA - https://www.facebook.com/groups/2077897069170052

This group is for information and education on the gene therapy treatment, Zolgensma.

RISDIPLAM (Evrysdi) Information for Spinal Muscular Atrophy - https://www.facebook.com/groups/383725645576828

This group is for those wanting to learn about and understand Risdiplam (commercial name is Evrysdi), a medical treatment taken by mouth or g-tube, once daily, for the treatment of SMA.

SPINRAZA Information for Spinal Muscular Atrophy - https://www.facebook.com/groups/SpinrazaInformationForSMA

This group is for those wanting to learn more about Spinraza, the first FDA-approved drug for the treatment of SMA delivered via intrathecal injection every 4 months.

Scholar Rock SRK-015 Info for SMA - https://www.facebook.com/groups/377854769923495

This is a group for those in the Scholar Rock SRK-015 trial to share information and for others to ask questions.

Annual SMA Conference

All individuals with SMA, families, and caregivers are invited to Cure SMA’s Annual SMA Conference! If you have any questions about the conference, please email [email protected].

Directions

This is Cure SMA’s publication for individuals with SMA and families.

Click here to view all of our editions of Directions.

Compass

This is Cure SMA’s publication that shares research updates.

Click here to view all of our editions of Compass.

FAQ Section

Are there clinical trials happening for SMA?
Yes. The following site has a list of all clinical trials that are associated with SMA, that individuals with SMA may qualify for: http://www.clinicaltrials.gov/ct2/results?term=Spinal+Muscular+Atrophy&Search=Search. You can also try out Cure SMA’s Clinical Trials Finder as well: https://clinicaltrials.curesma.org/search-results

For information about available clinical trials, go to www.clinicaltrials.gov and enter the following search terms: Spinal muscular atrophy.  Under other terms enter “adult” or “pediatric” (whichever is relevant to you) then enter the Country and complete additional questions. Once that information is filled in a list of clinical trials and participating centers will come up.

 

How can I learn about research happening for SMA treatment?
There is currently still research going on for SMA, with a few other clinical trials open and recruiting. Here is a link to our research page that talks about the drug pipeline and where each research program is in the pipeline: http://www.curesma.org/research/latest-advances/.

 

I am considering moving to the U.S. What kind of support can help me plan for my move?
If you are looking to relocate, please see the resources below:

If you do relocate to the U.S., please reach out to Cure SMA at [email protected] so that we can provide further information on support programs at the link above that you will be eligible for.

 

What is prenatal testing for SMA and how can it help?
Labs that offer prenatal diagnostic testing for SMA can report SMN2 copy number. SMN2 copy number is generally correlated with SMA type although there are exceptions. If you pursue prenatal testing for SMA, we suggest that you request SMN2 copy number if the SMA genetic testing (SMN1 copy number) is consistent with SMA.

SMA type is determined by age at which symptoms of SMA begin and highest developmental milestone achieved, so technically one cannot determine type or how SMA symptoms will present before birth. However, SMN2 copy number can provide an estimation of the level of motor function the baby would achieve if untreated.

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