Disease, Disorders, Medical Disciplines

Cure SMA

Make today a breakthrough.

aka Families of SMA

Elk Grove Village, IL

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

1985

President

Mr. Kenneth Hobby

Main Address

925 Busse Road

Elk Grove Village, IL 60007 USA

Keywords

spinal muscular atrophy, sma, research, families of sma, fsma

EIN

36-3320440

 Number

1138006102

Cause Area (NTEE Code)

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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Programs + Results

What we aim to solve

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 complex, multifaceted disease that demands action through a number of 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 families affected by SMA; and providing families with the practical support, education, and resources they need for today.

Our programs

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

SOURCE: Self-reported by organization

Programs

Where we work

Charting Impact

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

SOURCE: Self-reported by organization

What is the organization aiming to accomplish?

What are the organization's key strategies for making this happen?

What are the organization's capabilities for doing this?

How will they know if they are making progress?

What have they accomplished so far and what's next?

Cure SMA's ultimate aim is a world in which SMA is treatable and curable, and in which all those affected by SMA have access to the support and resources that they need to live active and engaged lives.
In December 2016, the FDA approved Spinraza, which treats the underlying cause of SMA. Spinraza is the first-ever FDA-approved therapy for SMA, a tremendous leap forward for those affected by the disease. And Spinraza is just the leading edge of a robust drug pipeline, with a breadth and depth that reflects a goal of treatments for all ages and types of SMA. In addition to the first-ever approved therapy, there have been many other advances in recent years, from new techniques in gene therapy, to drugs that affect muscle and nerve function and could be used in combination with drugs like Spinraza.
Cure SMA's aim is to move promising drug candidates toward clinical trials, and then to ensure that those clinical trials are conducted as efficiently as possible. Cure SMA also aims to ensure that effective treatments receive timely and appropriate regulatory approvals, as was the case with Spinraza.
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. Cure SMA is aiming to develop clinical evidence that will support the implementation of a standard of care for SMA, and also establish a network of SMA-focused clinical care centers across the US.
Cure SMA also aims to address issues that affect quality of life for those affected by SMA. This includes shortening the time to diagnosis, eliminating the physical, financial, and emotional costs of the “diagnostic odyssey" that many with SMA experience; advocating for robust insurance coverage for approved therapies, necessary clinical care, and durable medical equipment; and removing barriers, such as site capacity, that may prevent the timely delivery of life-saving treatments.
Finally, through our newly diagnosed outreach program, networking through local chapters, opportunities to participate in advocacy or fundraising, the Annual SMA Conference, and more, Cure SMA provides families with the daily support they need, and with opportunities to fight back against SMA.

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 affected receive the care and treatment they need in a number of 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 is able to 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. The goal is to have SMA added to the federal Recommended Uniform Screening Panel, and have that recommendation implemented in all 50 states.
Cure SMA has a full slate of family support services. Our newly diagnosed program provides information packets, care packages, wagons, and car beds to newly diagnosed families. We also maintain an equipment pool for items like medical strollers and feeder chairs.
Cure SMA hosts the Annual SMA Conference, bringing together researchers, healthcare professionals, and families to network, learn, and collaborate. Our conference is really two separate events—one for families, and one for researchers—that run in parallel, so that the whole community can interact with and learn from one another. The conference is the largest in the world focused specifically on SMA.
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.
Finally, Cure SMA has 34 chapters that focus on local advocacy, awareness, and fundraising events, and provide a forum for families affected by SMA 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. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data. The SMA Industry Collaboration works together to address scientific, clinical and regulatory topics that are critical for the broader SMA community.
The RFP process is overseen by the Cure SMA Scientific Advisory Board, a collection of the foremost SMA experts in the US. The SMA Industry Collaboration is governed by Cure SMA and a joint steering committee with representatives from all companies participating. Cure SMA's clinical care research and SMA clinical care center project 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 is one of the largest funders of SMA research, with nearly $70 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 largest SMA research conference (and largest patient conference) in the world.
Cure SMA has a full-time advocacy staff presence in Washington DC, and our 34 chapters provide a local presence in many key states.
Each year, Cure SMA reaches an estimated 3,500 to 4,000 families affected by SMA through a combination of our newly diagnosed outreach program, other family support efforts (such as the equipment pool), and the Annual SMA Conference. Cure SMA also hosts 250 to 300 fundraising events each year. In total, we have a network of over 115,000 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.

Cure SMA looks at a number of key metrics to determine our progress toward our goals.
One of the most important is the SMA drug pipeline, which lays out the number of programs in development, the different approaches being pursued, and the progress of each program toward FDA approval. Realizing that it often takes thousands of compounds to produce a single drug candidate for trials, and that only 10% of drugs that start clinical trials will ultimately receive approval, and realizing that it will take multiple drugs and multiple approaches to most effectively treat SMA—continuing to expand and develop this pipeline remains a primary focus.
Even with an approved therapy and with five additional drugs in clinical trials, continued funding for SMA research is critical to our ultimate goal of a world where SMA is treatable and curable. Cure SMA updates and evaluates the SMA drug pipeline twice per year.
Cure SMA also evaluates our impact through progress on other research initiatives, including regulatory engagement, expansion of SMA clinical trial networks, and SMA clinical care centers established.
Our newly diagnosed survey—which collects information from families and individuals at the time of diagnosis—and our yearly update survey—which collects information from all families and individuals affected by SMA—are both an important part of our research program, and a way to evaluate the impact of those programs. These surveys allow us to track outcomes such as the time to diagnosis, access to treatment, and impact of common SMA complications such as use of breathing support, contractures, and more.
Advocacy efforts are measured by favorable government policies implemented; funding received for newborn screening, NIH research and other areas; and number of states that implement newborn screening for SMA.
Our family support programs are evaluated by the number of families reached, both as a raw number, and as a percentage of expected annual cases based on birthrate and incidence. These programs not only provide support to families, but they provide an incentive to build a strong and unified community. This unified strength then allows us to make an impact in advocacy, fundraising, and data collection.
Finally, we also evaluate our impact via growth of the extended SMA community: those who are not personally impacted by the disease but have chosen to become supporters and partners with us.

To date, Cure SMA has funded $70 million dollars in research. This includes over 200 research grants, plus research through the SMA Industry Collaboration, and research conducted by Cure SMA's research staff. Cure SMA has funded over half of the ongoing novel drug programs for SMA. Beginning in 2003, Cure SMA provided the very first research funding needed to begin investigation into the therapeutic approach that led to Spinraza. Drs. Ravindra Singh and Elliot Androphy, through work funded by Cure SMA, identified the ISSN1 gene sequence, which is the sequence targeted in Spinraza. Spinraza was approved in three months, one of the fastest approvals in the history of the FDA, and was approved for individuals with all ages and types of SMA. Timely approvals and broad labeling were two of the primary goals of FDA advocacy efforts led by Cure SMA. Then, in April 2017, Cure SMA held a Patient Focused Drug Development (PFDD) Meeting with FDA leaders. During the meeting, those affected by SMA were invited to speak on the impact of SMA as well as their hopes for future treatments. This was only the second externally led PFDD meeting to be granted by the FDA. In 2007, Cure SMA funded the development of a standard of care paper for SMA. Cure SMA also funded and led the development of an updated standard of care paper, addressing new areas in response to the changing needs of those with SMA and the approval of new therapies. This updated standard of care is expected to be published in 2018. Cure SMA's Annual SMA Conference has grown from to the largest conference in the world focused on SMA. In 2017, a record 2,200 families and researchers came together. Cure SMA has also reached thousands of newly diagnosed families, loaned thousands of equipment pool items, and more. In 2017, prompted by the approval of Spinraza, Cure SMA submitted a nomination of SMA to the federal Recommended Uniform Screening Panel for newborn screening. A decision is expected in February 2018, at the completion of a nine-month evidence review process. Cure SMA's strategic goals for the next several years include: • Continuing to fund research, leading to the development, testing and approval of additional drugs, until we have effective treatments for all ages, types, and stages of SMA. • Expanding SMA clinical trial networks. Currently, there are more pivotal trials in SMA than at any point in history, and additional capacity is needed to make sure these trials are conducted safely and effectively. • Ensuring SMA newborn screening is implemented in all 50 states, as early treatment is required to achieve the best outcomes for those with SMA. • Establishing a network of SMA clinical care centers across the US, beginning with 8 sites in early 2018, and growing rapidly from there. Care centers will collect data to help validate and promote the standard of care for SMA, and provide those affected with SMA with access to the best care.

How We Listen

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

Source: Self-reported by organization

the feedback loop
check_box We shared information about our current feedback practices.
How is the organization collecting feedback?
We regularly collect feedback through: electronic surveys (by email, tablet, etc.), paper surveys, constituent (client or resident, etc.) advisory committees.
How is the organization using feedback?
We use feedback to: 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?
We share feedback with: the people we serve, our staff, our board, our funders, our community partners.
What challenges does the organization face when collecting feedback?
It is difficult to: we don't have any major challenges to collecting feedback.

External Reviews

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Financials

Cure SMA

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Operations

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

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Board Leadership Practices

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SOURCE: Self-reported by organization

BOARD ORIENTATION & 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?

Not Applicable

CEO OVERSIGHT

Has the board conducted a formal, written assessment of the chief executive within the past year?

Not Applicable

ETHICS & TRANSPARENCY

Have the board and senior staff reviewed the conflict-of-interest policy and completed and signed disclosure statements in the past year?

Not Applicable

BOARD COMPOSITION

Does the board ensure an inclusive board member recruitment process that results in diversity of thought and leadership?

Not Applicable

BOARD PERFORMANCE

Has the board conducted a formal, written self-assessment of its performance within the past three years?

Not Applicable