Better ways to measure symptoms and progress in LBSL
Project 3: Clinically Meaningful Outcomes in LBSL
This project uses wearable sensors and special brain scans to find meaningful ways to track symptoms and changes in people with LBSL.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11172781 on NIH RePORTER |
What this research studies
You and your family would be trained to use small wearable devices (OPAL) at home to record walking and balance. The team will collect remote gait and ataxia data and compare those readings with patient- and observer-reported outcomes to identify score changes that matter. You may also be invited to have proton MRS brain scans to measure lactate as a possible monitoring biomarker. Together these steps aim to create reliable outcome measures that can be used in future treatment trials.
Who could benefit from this research
Good fit: People with a confirmed diagnosis of LBSL who can wear home sensors, complete symptom reports, and attend scanning visits are ideal candidates.
Not a fit: Those without a confirmed LBSL diagnosis or who cannot use wearable devices or travel for MRS scans may not receive direct benefit from participation.
Why it matters
Potential benefit: If successful, the project could provide reliable, easy-to-collect measures to show whether a treatment is helping people with LBSL.
How similar studies have performed: Wearable gait measures and MRS lactate have shown promise in other neurological conditions, but applying them specifically to LBSL is a new and emerging approach.
Where this research is happening
Philadelphia, United States
- Children's Hosp of Philadelphia — Philadelphia, United States (Active)
Researchers
- Principal investigator: Fatemi, S. Ali — Children's Hosp of Philadelphia
- Study coordinator: Fatemi, S. Ali
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.