SMN-independent approaches for spinal muscular atrophy
Official title: Mechanisms and SMN-Independent Therapies for Spinal Muscular Atrophy
Funded at Columbia University Health Sciences · NIH-11307153 · Data updated 2026-11-12
This project looks for new treatments for people with spinal muscular atrophy that work without relying only on restoring the SMN protein.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | April 1, 2022 |
| Project end | March 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $484,338 |
| NIH project number | 5R01NS123292-05 |
| Project ID | NIH-11307153 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with spinal muscular atrophy—especially infants, young children, or patients who have not fully responded to SMN-targeting therapies—would be the main focus for eventual clinical testing.
✕Less likely to help
Patients with neurological conditions unrelated to SMA or those with very advanced, irreversible nerve and muscle damage are unlikely to benefit from the therapies under study.
What this research studies
This project studies why low SMN protein harms the nerves and muscles that control movement and tests therapies that do not depend on boosting SMN. Researchers use laboratory models, molecular studies, and gene-delivery tools such as AAV vectors to target other pathways that keep motor neurons and muscle healthy. The team aims to find treatments that could protect or restore function even in patients who do not fully respond to current SMN-focused drugs. Promising leads would be used to design future human studies and clinical trials.
Why it matters
Potential benefit: Could produce new treatments that help people with SMA, including those who gain limited benefit from current SMN-centered therapies.
How similar studies have gone: SMN-replacement therapies like Spinraza and AAV-based gene therapy have benefited many patients, but SMN-independent strategies are newer and less proven.
Where this research is happening
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: Monani, Umrao — Columbia University Health Sciences
- Study coordinator: Monani, Umrao
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.