Finding ways to improve metabolic issues in spinal and bulbar muscular atrophy
Official title: Therapeutic strategies to rescue metabolic deficiencies in spinal and bulbar muscular atrophy
Funded at Thomas Jefferson University · NIH-10826086 · Data updated 2026-11-12
This study is looking at a treatment that could help improve muscle function in adults with spinal and bulbar muscular atrophy (SBMA) by restoring important compounds in their muscles, using mice to see how it works.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Thomas Jefferson University NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | September 15, 2023 |
| Project end | August 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $429,000 |
| NIH project number | 1R21NS135773-01 |
| Project ID | NIH-10826086 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with spinal and bulbar muscular atrophy.
✕Less likely to help
Patients with other forms of muscular dystrophy or unrelated neurodegenerative diseases may not benefit from this research.
What this research studies
This research focuses on spinal and bulbar muscular atrophy (SBMA), a genetic neuromuscular disease that affects adults. The team is investigating how to restore important metabolic compounds in muscle tissue that are depleted in this condition. They are using mouse models to explore the effects of a specific treatment that aims to bypass a metabolic block, potentially leading to improved muscle function. By understanding the underlying mechanisms of SBMA, the researchers hope to develop effective therapeutic strategies.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that improve muscle function and quality of life for patients with spinal and bulbar muscular atrophy.
How similar studies have gone: While research on metabolic treatments for neurodegenerative diseases is ongoing, this specific approach using nicotinamide mononucleotide is relatively novel.
Where this research is happening
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Thomas Jefferson University
Philadelphia, United States Active
Researchers
- Principal investigator: Merry, Diane E — Thomas Jefferson University
- Study coordinator: Merry, Diane E
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.