Cure SMA

Make today a breakthrough.

aka Families of SMA   |   Elk Grove Village, IL   |  http://www.curesma.org

Mission

Cure SMA leads the way to a world without spinal muscular atrophy, the number one genetic cause of death for infants. We fund and direct comprehensive research that drives breakthroughs in treatment and care, and we provide families the support they need for today.

Ruling year info

1985

President

Mr. Kenneth Hobby

Main address

925 Busse Road

Elk Grove Village, IL 60007 USA

Show more contact info

EIN

36-3320440

NTEE code info

Specifically Named Diseases (G80)

Nerve, Muscle and Bone Diseases (G50)

Birth Defects, Genetic Diseases Research (H20)

IRS filing requirement

This organization is required to file an IRS Form 990 or 990-EZ.

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Communication

Programs and results

What we aim to solve

SOURCE: Self-reported by organization

Spinal muscular atrophy (SMA) is a disease that robs people of 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. SMA affects approximately 1 in 11,000 babies, and about 1 in every 50 Americans is a genetic carrier, though most are unaware that they are carriers until they have a child with SMA. SMA is a complex, multifaceted disease that demands action through several avenues. This includes funding and directing research that will lead to breakthroughs in treatment and care; advocating with government, payers, and regulators for the issues that impact individuals with SMA and their families; and providing the community with the practical support, education, and resources they need for today.

Our programs

SOURCE: Self-reported by organization

What are the organization's current programs, how do they measure success, and who do the programs serve?

Programs

To date Cure SMA has raised and invested over $70 million towards SMA research to discover and develop a treatment and cure for SMA. Cure SMA sponsors the leading annual global conference devoted entirely to SMA research, the International Spinal Muscular Atrophy Research Group Meeting. Researchers gather from around the world to share results and exchange ideas.
A conference for families is held in conjunction with the research conference - for families to learn about the status of SMA research, share the latest in disease management techniques, and network with other families.
We publish a newsletter, Directions, quarterly, and we have an interactive web site for families to access the latest information and support each other. We also publish a quarterly research publication "Compass" to update members and donors on SMA research and clinical trials. Cure SMA maintains a web site and forums to create a ongoing knowledge base. Our 800 # is available 24 hours a day for emergency support. Cure SMA also loans equipment.

Population(s) Served
Adults

Where we work

Goals & Strategy

SOURCE: Self-reported by organization

Learn about the organization's key goals, strategies, capabilities, and progress.

Charting impact

Four powerful questions that require reflection about what really matters - results.

Cure SMA's aim is a world in which SMA is treatable and curable, and in which all those with SMA have access to the support and resources that they need to live active and engaged lives.

The research Cure SMA funds continues to expand our knowledge of SMA and deliver treatment opportunities, as reflected in the progress seen with Evrysdi, Spinraza, and Zolgensma. With these three treatments for SMA now approved by the U.S. Food and Drug Administration (FDA), we are now advancing new research that focuses on non-SMN targeted therapies, symptom and muscle-targeting treatments, and the development of combination therapies that increase strength and enable independence for all people with SMA.

Early diagnosis and early treatment of SMA is critical. Currently, 7 in 10 babies born in the U.S. are screened for SMA at birth and the country is on track to exceed screening of 90 percent of all births by 2022. This will lead to early diagnosis and then early treatment, and ultimately better outcomes. Cure SMA will continue working with all non-screening states to implement newborn screening for SMA to ensure babies in every state have the best chance at a healthy and full life.

As approved treatments extend lifespan, the SMA population will increase, with more individuals alive and living longer with SMA. This means that proper care is more vital than ever before. With new information from our SMA Clinical Data Registry, we are improving our understanding of the best care for SMA. By following patient outcomes and treatment progression, we will create an evidence-based standard of care for SMA. This requires continued growth and integration of the Cure SMA Care Center Network to ensure a comprehensive registry and improved local patient care.

Advocacy remains a core tenant of Cure SMA’s strategy. We continue efforts to educate public and private insurers on the ongoing needs of the SMA community and the importance of access to life-altering medications. Additionally, Cure SMA looks to accelerate our work on policy issues most important to adults and young adults with SMA.

Finally, Cure SMA aims to address issues that affect quality of life for those with SMA and their families. As SMA changes, so must our efforts to unite local communities through Cure SMA Chapters, support programs including Summits of Strength, and community events like Walk-n-Rolls. With a greater local presence, we can ensure access to approved treatments and care, and we can create customized programs that meet the new needs of everyone with SMA in local communities.

Cure SMA supports research in a variety of areas. This includes funding basic research to investigate the biology of SMA and reveal new and more effective ways of making drugs for SMA; drug discovery research, which converts those basic research ideas into drug candidates that can be tested in clinical trials; and clinical and regulatory research, which enables effective drug candidates to move efficiently through trials and regulatory approval. These three phases of research support a robust pipeline of SMA drug programs, offering both a depth of options and a breadth of approaches. Research suggests that it will take a combination of drugs that work in different ways to most effectively treat SMA. Cure SMA also supports research and implementation of a standard of care for SMA, so that individuals can receive the care and treatment they need in several areas, including breathing, nutrition, and musculoskeletal issues.

Cure SMA maintains the largest patient database for SMA in the world. Through focus groups, surveys, and a yearly database update, Cure SMA can demonstrate the impact of SMA over time, leading to data that can improve outcomes for those with SMA, raise awareness of SMA in the research community, and increase understanding of the burden of SMA.

Cure SMA actively advocates at the federal and state level for policies favorable to our community. This includes funding for multiple programs and projects, including Medicare and Medicaid, and research through the NIH. A particular focus of Cure SMA's current advocacy is newborn screening. Since SMA was added to the federal Recommended Uniform Screening Panel, our goal has been to have screening for SMA implemented in all 50 states by 2022.

Cure SMA has a full slate of family support services. Our newly diagnosed program provides information packets, care packages, and useful equipment (e.g., wagons, car beds, etc.) to newly diagnosed families. We also maintain an equipment pool for items like medical strollers and feeder chairs, as well as offer a care package for teens and adults with products that allow them to find more independence.

Cure SMA hosts the Annual SMA Conference, bringing together researchers, healthcare professionals, individuals with and families to network, learn, and collaborate. Newly diagnosed families are given a scholarship to the Annual SMA Conference and invited to attend a special newly diagnosed program. Cure SMA also offers financial need scholarships and scholarships for adults with SMA.

The Annual SMA Conference runs in parallel to the SMA Research & Clinical Care Meeting, which review relevant research and care protocols with the scientific and provider communities. Both events are the largest in the world focused specifically on SMA.

Finally, Cure SMA has 36 chapters that focus on local advocacy, awareness, and fundraising events, and provide a forum for individuals with SMA and their families to learn from and offer support to each other.

Cure SMA's research portfolio is executed through a combination of grants awarded by Cure SMA through a competitive RFP process, direct research conducted by Cure SMA staff, and research through a collaborative industry group led by Cure SMA.

Cure SMA is one of the largest funders of SMA research, with more than $82 million funded thus far, and is recognized as a leader in the research and clinical communities, with resources including the largest SMA patient database in the world (the SMA Clinical Data Registry), a network of SMA designated treatment sites (the Cure SMA Care Center Network), and the largest SMA research conference in the world.

The Cure SMA Industry Collaboration was established in 2016. It leverages the experience, expertise, and resources of pharmaceutical, biotechnology companies, and other nonprofit organizations involved in the development of SMA therapeutics to address a range of scientific, clinical, and regulatory challenges more effectively.

The RFP process for Cure SMA grants is overseen by the Cure SMA Scientific Advisory Board, a collection of the foremost SMA experts in the U.S. Research and clinical projects are overseen by the Medical Advisory Council, a group of clinicians and experts covering multiple areas, including neurology, pulmonology, physical and occupational therapy, nutrition, and more.

Cure SMA has a full-time advocacy staff presence in Washington D.C., and volunteer advocates from across the country provide a local presence in many key states.

Each year, Cure SMA reaches an estimated 10,000+ individuals with SMA and their families through a combination of our newly diagnosed outreach program, other family support efforts (such as the equipment pool), virtual and in-person events like the Summits of Strength, and the Annual SMA Conference. In total, we have a network of over 120,000 families and supporters.

Cure SMA's rapidly growing staff team includes 35 employees with expertise in all areas. And, Cure SMA's key staff leaders each have decades of experience in their respective fields. Cure SMA is governed by an 18-member Board of Directors, and 14 committee members.

To date, Cure SMA has achieved the following milestones:
• Invested more than $82 million in research funding in support of over 80 basic and drug discovery research programs, which have led to three FDA-approved treatments for SMA.
• Established 19 Cure SMA Care Center Network site across the country.
• Implemented newborn screening for SMA in 33 states, representing approximately 68 percent of babies born in the U.S.
• Grew the SMA Clinical Data Registry to include approximately 380 patients, representing a diverse cross-section of individuals with SMA in the U.S.
• Built a community of more than 120,00 individuals with SMA, their families and friends, researchers, and healthcare providers that support a stronger future for people with SMA.
• Provided educational materials, care packages, and needed equipment to tens of thousands of people connected with the SMA community.

Cure SMA's strategic goals for the coming years include:
• Continuing to fund research, leading to the development, testing and approval of additional treatments for SMA for all ages, types, and stages of SMA.
• Expanding the Cure SMA Care Center Network to support clinical trial recruitment and ensure the treatment needs of the community are met.
• Ensuring SMA newborn screening is implemented in all 50 states, as early treatment is required to achieve the best outcomes for those with SMA.
• Advocating with state and federal lawmakers to ensure greater access to products and services for people with disabilities.
• Building on the data collected through the SMA Clinical Data Registry to help validate and promote a standard of care for SMA and provide those with SMA access to the best care and outcomes possible.
• Providing support programs for individuals with SMA and their families, including both live and virtual options. This includes expanding current programming and offering new.

How we listen

SOURCE: Self-reported by organization

Seeking feedback from people served makes programs more responsive and effective. Here’s how this organization is listening.

done We shared information about our current feedback practices.
  • How is your organization collecting feedback from the people you serve?

    Electronic surveys (by email, tablet, etc.), Paper surveys, Constituent (client or resident, etc.) advisory committees, Suggestion box/email,

  • How is your organization using feedback from the people you serve?

    To identify bright spots and enhance positive service experiences, To make fundamental changes to our programs and/or operations, To inform the development of new programs/projects, To strengthen relationships with the people we serve,

  • With whom is the organization sharing feedback?

    The people we serve, Our staff, Our board, Our funders, Our community partners,

  • Which of the following feedback practices does your organization routinely carry out?

  • What challenges does the organization face when collecting feedback?

    We don't have any major challenges to collecting feedback,

Financials

Cure SMA
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Operations

The people, governance practices, and partners that make the organization tick.

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Connect with nonprofit leaders

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  • Analyze a variety of pre-calculated financial metrics
  • Access beautifully interactive analysis and comparison tools
  • Compare nonprofit financials to similar organizations

Want to see how you can enhance your nonprofit research and unlock more insights? Learn More about GuideStar Pro.

Cure SMA

Board of directors
as of 3/26/2021
SOURCE: Self-reported by organization
Board chair

Mr. Nick Farrell

Bradley Nunemaker

Corey Braastad, PhD

Annie Kennedy

Spencer Perlman

Shannon Zerzan

Peter Statile

Edmund Lee

Matthew Evans

Brian Snyder

Jaclyn Greenwood

Tom Murray

Shannon Shryne

Kelly Cole

Gillian Mullins

Robert Graham

Allyson Henkel

Kelly Jankowski

Rob Lockwood

Amy Medina

Brynne Willis

Board leadership practices

SOURCE: Self-reported by organization

GuideStar worked with BoardSource, the national leader in nonprofit board leadership and governance, to create this section.

  • Board orientation and education
    Does the board conduct a formal orientation for new board members and require all board members to sign a written agreement regarding their roles, responsibilities, and expectations? Yes
  • CEO oversight
    Has the board conducted a formal, written assessment of the chief executive within the past year ? Yes
  • Ethics and transparency
    Have the board and senior staff reviewed the conflict-of-interest policy and completed and signed disclosure statements in the past year? Yes
  • Board composition
    Does the board ensure an inclusive board member recruitment process that results in diversity of thought and leadership? Yes
  • Board performance
    Has the board conducted a formal, written self-assessment of its performance within the past three years? Yes

Organizational demographics

SOURCE: Self-reported; last updated 1/22/2021,

Who works and leads organizations that serve our diverse communities? GuideStar partnered on this section with CHANGE Philanthropy and Equity in the Center.

Leadership

The organization's leader identifies as:

Race & ethnicity
White/Caucasian/European
Gender identity
Male

Race & ethnicity

No data

Gender identity

No data

 

No data

Sexual orientation

No data

Disability

No data