StudySMA Real World Study
This observational study collects real-world health data from individuals living with Spinal Muscular Atrophy (SMA) to better understand treatment patterns, disease progression, and long-term outcomes. By securely sharing your medical records, you help advance research that can shape the future of SMA care — from newborn screening through adulthood.
Participate from Home
No blood draws, procedures, or in-person visits
Securely share your EHR data to advance SMA research within 5 minutes
You may be eligible if you:
- Have been diagnosed with or are suspected of having Spinal Muscular Atrophy (Types 1, 2, 3, or 4)
- Are any age (newborn through adult)
- Have any treatment status (currently treated, previously treated, or treatment-naive)
- Are able to provide informed consent (or parental consent for minors)
About this research study
Spinal Muscular Atrophy (SMA) is a rare genetic disease caused by mutations in the SMN1 gene. With three FDA-approved therapies now available — and newborn screening programs expanding nationwide — the SMA treatment landscape is evolving rapidly. This study helps capture how these changes are playing out in real-world clinical practice, giving researchers the evidence they need to improve care and outcomes for the SMA community.
Our Approach
In partnership with the Muscular Dystrophy Association (MDA), this study uses Unite’s platform to securely collect and analyze structured medical data and unstructured clinical notes from your health records. This captures rich details — like treatment rationale and motor function assessments — that traditional registries often miss.
What We Study
We track real-world outcomes across approved SMA therapies including nusinersen (Spinraza), onasemnogene (Zolgensma), and risdiplam (Evrysdi). We analyze biomarker trajectories, motor function scores (HFMSE, RULM, CHOP-INTEND), treatment switching patterns, and the impact of newborn screening on early treatment initiation.
Built around one rule: it’s your data.
You’re in Control
You choose which records to share, and who to share them with. Only you can grant and revoke access. Unite will never sell your data.
Hospital-grade security
Your data is protected by end-to-end encryption and stored on secure, regularly audited servers. Regular audits and access controls keep your information safe — applied to everything we do.
Always free, no exceptions
Accessing your records, connecting your providers, and participating in research is completely free. No premium tiers, no paywalls — ever.
Take control of your health story today
Your medical records should make your life easier, not harder. Unite brings them together— and makes them work for you.
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