Understanding how muscle degeneration occurs in spinal and bulbar muscular atrophy (SBMA)
Mechanisms of neuromuscular degeneration in SBMA
['FUNDING_R01'] · UNIVERSITY OF MICHIGAN AT ANN ARBOR · NIH-11116863
This study is looking into how a genetic change in the androgen receptor causes muscle weakness in people with spinal and bulbar muscular atrophy (SBMA), and it hopes to find new ways to help manage the condition using special treatments tested in mice.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF MICHIGAN AT ANN ARBOR (nih funded) |
| Locations | 1 site (ANN ARBOR, UNITED STATES) |
| Trial ID | NIH-11116863 on ClinicalTrials.gov |
What this research studies
This research investigates the mechanisms behind spinal and bulbar muscular atrophy (SBMA), a condition that leads to muscle weakness due to a genetic mutation in the androgen receptor gene. The study focuses on how this mutation causes toxic effects in skeletal muscle, which may lead to the degeneration of motor neurons over time. Researchers will use specially designed mice to test their hypothesis and explore the role of antisense oligonucleotides to potentially mitigate the effects of the genetic mutation. By understanding these processes, the research aims to uncover new therapeutic strategies for SBMA.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with spinal and bulbar muscular atrophy, particularly those experiencing early symptoms.
Not a fit: Patients with other forms of muscular dystrophy or neuromuscular disorders unrelated to SBMA may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that slow down or prevent muscle degeneration in patients with SBMA.
How similar studies have performed: Previous research has shown promising results in understanding the mechanisms of neuromuscular degeneration, but this specific approach using antisense oligonucleotides in SBMA is relatively novel.
Where this research is happening
ANN ARBOR, UNITED STATES
- UNIVERSITY OF MICHIGAN AT ANN ARBOR — ANN ARBOR, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LIEBERMAN, ANDREW P — UNIVERSITY OF MICHIGAN AT ANN ARBOR
- Study coordinator: LIEBERMAN, ANDREW P
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 below.