Understanding brain changes in Duchenne muscular dystrophy
Cerebellar pathology in the absence of plasticity gating
This study is looking at how a lack of a protein called dystrophin affects the brain, especially in people with Duchenne muscular dystrophy, and it aims to understand how this might lead to problems with movement and learning, so we can find ways to help improve brain function for those affected.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Texas Hlth Science Center NIH-funded |
| Lab location | 1 site (San Antonio, United States) |
| Project ID | NIH-11087573 on NIH RePORTER |
What this research studies
This research investigates how a deficiency in dystrophin, a protein crucial for muscle function, affects the brain, particularly the cerebellum, in patients with Duchenne muscular dystrophy (DMD). The study focuses on the role of cerebellar Purkinje cells, which are important for motor control and learning, and examines how weakened synaptic connections and impaired plasticity may contribute to neurological symptoms in DMD. By using advanced techniques to measure brain activity, the researchers aim to identify potential therapeutic strategies that could restore normal brain function in affected individuals.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Duchenne muscular dystrophy who may experience neurological symptoms.
Not a fit: Patients without Duchenne muscular dystrophy or those whose neurological symptoms are unrelated to dystrophin deficiency may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that improve neurological symptoms in patients with Duchenne muscular dystrophy.
How similar studies have performed: Previous research has shown promising results in understanding the neurological impacts of dystrophin deficiency, suggesting that this approach could yield valuable insights.
Where this research is happening
San Antonio, United States
- University of Texas Hlth Science Center — San Antonio, United States (Active)
Researchers
- Principal investigator: Pugh, Jason Richard — University of Texas Hlth Science Center
- Study coordinator: Pugh, Jason Richard
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.