SMA Industry Collaboration
No single group can research and develop a treatment for spinal muscular atrophy (SMA) alone—it requires collaboration between academics, industry, government, and the SMA community. We bring all those groups together and lead the SMA Industry Collaboration to advance the goals that are important to our SMA community.
One of the most distinctive aspects of Cure SMA’s research program is the SMA Industry Collaboration. Established in 2016, 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 to advancing drug development in SMA and will benefit the broader SMA community. It is currently comprised of our partners at Biogen, Novartis, Scholar Rock, Genentech/Roche Pharmaceuticals, argenx, NMD Pharma, and SMA Europe.
Through the SMA Industry Collaboration, we fund research 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 research ensures 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. The SMA Industry Collaboration also conducts research and develops initiatives to identify and address barriers that limit the SMA community's access to clinical research, treatment, and care.
The industry collaboration currently consists of four topic groups:
Approaches to enabling drug development and clinical trials
Cure SMA Patient and Caregiver-Reported Data
Real-World Healthcare Provider Data on SMA Treatment and Supportive Care
Education and Engagement on SMA Treatment and Supportive Care
Current and Past SMA Industry Collaboration Projects
Engaging the FDA to educate on risk tolerance, treatment experiences, and unmet needs in the SMA community.
Development of resources to improve clinical trial awareness, knowledge, and access among the SMA community.
Conducting clinical trial readiness and education initiatives to increase the number of sites that can conduct SMA clinical trials. This includes toolkits for clinical trial coordinators and physical therapies.
Collaboration with SMA Europe to create resources and initiatives for the expansion of SMA clinical trial sites in Europe and impacted individuals and families residing in the region.
Development and refinement of outcome measures to ensure that SMA clinical trials are measuring the results most important to those with SMA.
Launching an annual Community Update Survey to better understand the impact of SMA and how the needs of the community change as treatments are made available.
Development of a quarterly newsletter for healthcare providers to enhance awareness of the latest updates in SMA treatment and care.
Educating public and private insurers on the unmet needs of individuals with SMA and barriers that limit community access to essential treatments and care.
Offering SMArt Moves, an education and awareness campaign to reduce the time to diagnosis so babies with SMA can get the most benefit from approved treatments.
Conducting a Critical Path Innovation Meeting (CPIM) with the FDA to discuss the community’s ongoing unmet medical needs.
Development of a competency-based formal education program for SMA physical therapists.
Evaluation of opportunities to “bring trials to patients” via the adoption of telemedicine and remote monitoring.
Holding a Patient-Focused Drug Development Meeting with the FDA and publishing a Voice of the Patient report based on those findings..
SMA Industry Collaboration Publications
Publishing our work allows us to share ideas and work collaboratively to advance SMA drug development. Access manuscripts, toolkits, and other resources generated by the Industry Collaboration.
SMA Industry Collaboration Latest News
SMA Community Risk Tolerance Update: Comparison of 2022 and 2017 SMA Risk/Benefit Survey Data: https://www.curesma.org/sma-community-risk-tolerance-update-comparison-of-2022-and-2017-sma-risk-benefit-survey-data/
Check Out Cure SMA’s 2024 State of SMA Report: https://www.curesma.org/2024-state-of-sma-report/
Last Chance to Participate in the Annual Cure SMA Community Update Survey: https://www.curesma.org/last-chance-to-participate-in-the-annual-cure-sma-community-update-survey/
Cure SMA Educates Insurers on Unmet Needs and Access Barriers of the SMA Community: https://www.curesma.org/cure-sma-educates-insurers-on-unmet-needs-and-access-barriers-of-the-sma-community/
Cure SMA Shares Patient Voice and Needs with FDA: https://www.curesma.org/cure-sma-shares-patient-voice-and-needs-with-fda/
Cure SMA Shares Updates on the Phase 10 SMA Industry Collaboration: PENDING PUBLICATION
Participate in the Annual Cure SMA Community Update Survey – Open Now: https://www.curesma.org/2024-cure-sma-community-update-survey-open-now/
Cure SMA Announces Expanded Phase 9 of SMA Industry Collaboration: https://www.curesma.org/cure-sma-announces-expanded-phase-9-of-sma-industry-collaboration/
Cure SMA Launches New Resources to Enhance Clinical Trial Access: https://www.curesma.org/cure-sma-launches-new-resources-to-enhance-clinical-trial-access/
Cure SMA Attends Spring 2024 Professional Organization Conferences to Promote Findings from Industry Collaboration Initiatives: https://www.curesma.org/cure-sma-attends-spring-2024-professional-organization-conferences-to-promote-findings-from-industry-collaboration-initiatives/
Check Out Cure SMA’s 2023 State of SMA Report: https://www.curesma.org/check-out-cure-smas-2023-state-of-sma-report/
- 01/26/2023 - Cure SMA Supports Clinical Trial Readiness
- 04/04/2023 - Cure SMA Announces Expanded Phrase 8 of SMA Industry Collaboration
- 07/12/2023 - Cure SMA's 2022 State of SMA Report
- 12/20/2023 - Analysis: Cure SMA's Risk/Benefit Survey
- 10/04/2022 - Cure SMA Holds Patient-Led Listening Session with FDA
- 09/08/2022 - Cure SMA Launches Benefit-Risk Survey to Assess Patient Perspective on Treatment Possibilities and Risks
- 08/15/2022 - Cure SMA Publishes Results from Survey Measuring Disease Burden in Adolescents and Young Adults
- 08/15/2022 - Cure SMA Publishes Research Exploring What Is Important to SMA Clinical Trial Participants
- 08/01/2022 - Cure SMA to Hold Patient-Led Listening Session with FDA
- 06/09/2022 - Cure SMA Attends Spring 2022 Conferences to Promote Findings from Industry Collaboration Initiatives
- 03/23/2022 - Cure SMA Announces Expanded Phase 7 of SMA Industry Collaboration
- 12/2/2021 – Survey Alert: SMA Family Caregivers
- 05/28/2021 – Cure SMA Published Manuscript on SMA Awareness, Screening, and Referral Patterns Amongst Pediatricians
- 04/19/2021 – 2021 Annual Cure SMA Community Update Survey
- 04/22/2021 – Cure SMA Attends Spring 2021 Conferences to Promote Findings from Industry Collaboration Initiatives
- 03/11/2021 – Cure SMA Publishes Quality of Life in Teens and Young Adults Manuscript in Orphanet Journal of Rare Diseases
- 12/21/2020 – Cure SMA Funds Over $9 Million in New and Ongoing Research in FY20
- 12/14/2020 – Cure SMA, Novartis Publish Economic Burden of SMA Analysis in Journal of Market Access & Health Policy
- 06/02/2020 – Cure SMA Publishes Article on SMA Clinical Trial Readiness Program in the U.S.
- 03/19/2020 – Cure SMA Launches Part 2 of “Spotlight on SMA” in Partnership With Neurology Reviews
- 02/11/2020 – Cure SMA Launches “Spotlight on SMA” Partnership With Neurology Reviews
- 12/19/2019 – A Look Back at 2019: Cure SMA Research Year-in-Review
- 11/27/2019 – Check Out the Cure SMA Booklet on “Scientific Considerations for Drug Combinations”
- 05/14/2019 – Cure SMA Publishes Journal Article on Benefit-Risk Preferences in SMA
- 01/02/2019 – Cure SMA Announces Expanded Phase 3 of SMA Industry Collaboration