Cure SMA Announces Expanded Phase 8 of SMA Industry Collaboration

Cure SMA is pleased to announce the launch of an expanded Phase 8 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, SMA Europe, Cure SMA, and other nonprofit organizations to share information, ideas, and data to benefit the broader SMA community. Through the Industry Collaboration, we fund projects to ensure that effective, safe treatments can progress through clinical trials quickly and gain approval from the U.S. Food and Drug Administration (FDA) and international regulators. Our work also ensures that these treatments address the unmet needs of the SMA community, and that the community’s priorities and goals are incorporated into the development, review, and approval of therapies. Click here to see what the SMA Industry Collaboration achieved during Phase 7!

As the SMA Industry Collaboration embarks on its eighth year, Cure SMA and our partners at Biogen, Scholar Rock, Novartis Gene Therapies, Biohaven Pharmaceuticals, Epirium Bio, Genentech/Roche Pharmaceuticals, and SMA Europe will build upon previous achievements to further refine treatment outcome measures, improve clinical trial site capacity, and enhance knowledge of the SMA disease experience and the needs of affected individuals. Additionally, we will work to identify and address barriers that limit the SMA community’s access to clinical research, treatment, and care.

The SMA Industry Collaboration is sub-divided into “Topic Groups” that each have a particular focus and specific goals. Every year, each Topic Group outlines the projects it will complete to achieve those goals. This year, the collaboration has added a fourth topic group, which will be led by the advocacy organization, SMA Europe, to support the needs of the European SMA community.

Below is a description of the Phase 8 goals and priorities of each SMA Industry Collaboration Topic Group.

Topic Group 1: Approaches to Enabling Drug Development and Clinical Trials


  • To enhance SMA clinical trials by developing healthcare provider training resources and improving treatment outcome measures.
  • To engage regulatory authorities and deepen their understanding of SMA and the perspectives of SMA-affected individuals on which treatment outcomes are clinically meaningful.

Phase 8 Projects

  • Understanding the SMA Communities Perspectives on Treatment Benefits and Risks

In 2017, Cure SMA conducted the first ever benefit/risk survey of the SMA community. To reevaluate the community's perspectives on treatment options and side effect tolerance within the shifting therapeutic landscape, Cure SMA repeated the same benefit/risk survey in 2022. This year, we will perform a comparative analysis of the 2017 and 2022 results to determine statistically significant changes in community perspectives. Results will be published and shared with the SMA community and FDA.

  • Improving Outcome Measures for Adults

Topic Group 1 will continue to develop and improve outcome measures for adults with SMA by developing and validating the following in collaboration with the Pediatric Neuromuscular Clinical Research Network (PNCRN):

    • The Adapted Test of Neuromuscular Disorders (ATEND), a scale that measures wheelchair-based motor function scale.
    • Bulbar assessments of voice, speech, and swallow.
    • The SMA Personal Reported Outcome Measure (SMA-PRO), the goal of which is to broaden the scope of the PEDI-CAT to better meet the needs of SMA-affected individuals across all ages and phenotypes.
    • The SMA Functional Composite Score (SMA-FC), which is a combination of the HFMSE, RULM, and 6MWT. The group wants to determine how well the SMA-FC measures changes the treated SMA populations over time compared to a single assessment.


  • Assessing Challenges in Clinical Trial Recruitment

SMA clinical trials continue to face barriers to developing trial sites and recruiting participants. We will assess how changes in the screening landscape, staffing shortages, and facility constraints have affected site capacity and recruitment potential since Cure SMA launched the Clinical Trial Readiness Program in 2018. We will then revamp the program to address the ongoing needs of research staff and expand the number of potential trial sites.

Topic Group 2: Education and Awareness to Promote Broad and Inclusive Research, Treatment, and Care of SMA Patients


  • To engage individuals and families affected by SMA to understand and identify disparities in SMA care, treatment, and clinical trial research.

Phase 8 Project

  • Identify Barriers to Clinical Trial Participation

This year, Cure SMA will conduct a survey for adults living with SMA and caregivers to better understand the barriers that impact the communities access to SMA clinical research. The survey will build upon the findings of the Community Access Discussion Groups conducted by the topic group during the previous year.  It will explore the factors that affect enrollment, as well as the community’s perspectives on the availability of clear and relatable information regarding SMA clinical trials.

  • Enhance Community Awareness and Access to SMA Clinical Trials

Topic Group 2 will use the findings obtained from the above survey, to develop a campaign to increase clinical trial awareness, knowledge, and access among the SMA community. Campaign materials will be designed to engage and inform individuals living with SMA, caregivers, and providers about the drug development and clinical trial processes so that they can make informed decisions about whether clinical trial participation is right for them.

Topic Group 3: Cure SMA Patient-Reported Data


  • To develop tools to collect patient-reported data that will help us better understand the natural history of SMA, and the evolution of the SMA disease experience as new therapies are developed.

Phase 8 Project

  • The 7th Annual Cure SMA Community Update Survey

Since the initial launch in 2017, the Cure SMA Community Update Survey has collected data and information on the SMA community’s experiences and daily challenges. Our top priority is to represent the patient voice from the whole community, so we can drive research and care to meet patient needs. Every piece of data collected allows Cure SMA to track changes in the attitudes, feelings, and actions of the SMA community over time. Cure SMA is currently working to analyze five years of data captured via the Annual Community Update Survey to better understand changes in the SMA disease experience. During Phase 8, Cure SMA will also analyze survey data to identify healthcare disparities within the SMA community and understand how the social determinants of health (such as housing and food security, income and primary language spoken at home) affect access to treatment. These efforts will guide the development of resources for the SMA community including families, clinicians, and researchers. The 7th Annual Community Update Survey will be distributed in the spring of 2023.

Topic Group 4: SMA Europe


  • To enhance SMA clinical trials in Europe by engaging individuals and families affected by SMA to understand disparities impacting clinical trial participation and support the development of provider training resources.

Phase 8 Project

  • Optimizing the Clinical Trial and Treatment Readiness Hub

Topic Group 4 will survey SMA-affected adults and caregivers about the factors that may impact their willingness to participate in clinical trials. The questionnaire will be distributed across European countries with the support of SMA Europe’s national member organizations. Additionally, SMA Europe will engage with European clinical trial sites to obtain insight on capacity and trial recruitment challenges. The information obtained from both initiatives will be leveraged by SMA Europe to optimize Clinical Trial and Treatment Readiness Hub resources.

SMA Industry Collaboration Phase 7 (2022) Achievements

During Phase 7, the SMA Industry Collaboration spearheaded research efforts that increased SMA treatment awareness and addressed the unmet needs of the SMA community. A summary of these accomplishments may be found in the 2022 End of Year Report.

The SMA Industry Collaboration’s Phase 7 Accomplishments include:

  • Publishing five research articles in peer-reviewed journals
  • Hosting a Patient-Led Listening Session with the FDA to give members of the SMA community a forum to offer their perspectives on current and future SMA therapeutics
  • Conducting focus group activities for affected adults and caregivers to identify barriers that limit access to research, treatment, and care in the SMA patient community
  • Distributing a survey to understand our community’s perspectives on treatment possibilities and tolerance for the side effects of currently available and planned therapies
  • Developing outcome measures designed for teens and adults (SMA-PRO, ATEND, SMACR-HI)
  • Developing assessments for voice, speech, and swallow
  • Conducting discussion groups to better understanding caregiver perspectives on patient-reported outcome (PRO) research instruments
  • Distributing a survey to acquire comprehensive demographic information for individuals and families affected by SMA
  • Producing a video series to educate providers on the importance of SMA screening, diagnosis of SMA in children and adolescents through telehealth, and conducting telehealth appointments with patients


About the Cure SMA Industry Collaboration

The Cure SMA Industry Collaboration was established in 2016 to leverage the experience, expertise, and resources of pharmaceutical, biotechnology companies, and other nonprofit organizations involved in the development of spinal muscular atrophy (SMA) therapeutics to more effectively address a range of scientific, clinical, and regulatory challenges. It is currently comprised of our partners at Biogen, Scholar Rock, Novartis Gene Therapies, Biohaven Pharmaceuticals, Epirium Bio, Genentech/Roche Pharmaceuticals, and SMA Europe.



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