Understanding early Friedreich ataxia in children to improve how we measure outcomes
Official title: Understanding early natural history to facilitate new clinical outcome assessments and trial design in children with Friedreich Ataxia (FRDA)
Funded at Children's Hosp of Philadelphia · NIH-11494983 · Data updated 2026-11-12
This study looks at how Friedreich ataxia starts and progresses in kids to create better, child-friendly measures for tracking the disease and testing treatments.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | September 1, 2026 |
| Project end | August 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $576,029 |
| NIH project number | 1R01FD008864-01 |
| Project ID | NIH-11494983 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and young adults with Friedreich ataxia who are within the early disease course and can participate in a 24-month follow-up with biomarker and digital outcome assessments.
✕Less likely to help
Patients with advanced FRDA or those unable to participate in digital health or blood biomarker procedures may have limited direct benefit.
What this research studies
Friedreich ataxia is a rare, childhood-onset neurodegenerative disease that worsens over time. The EARLY-FA project will enroll 125 children and young adults with FRDA to map the early disease course over two years using digital health tools and new biomarkers. Researchers will study how growth, puberty, body composition, and FRDA features relate to outcomes and identify blood-based biomarkers that reflect disease severity. They will also test new digital measures of ataxia to capture functional changes more precisely. This work will also utilize data from a larger global natural history effort (UNIFAI) to strengthen understanding of early FRDA.
Why it matters
Potential benefit: If successful, the findings could enable earlier, more accurate tracking of disease progression and faster evaluation of new therapies in children with FRDA.
How similar studies have gone: Previous natural history studies and biomarker work in FRDA support the feasibility of this approach, though the early pediatric focus is relatively novel.
Where this research is happening
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Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Lynch, David Robinson — Children's Hosp of Philadelphia
- Study coordinator: Lynch, David Robinson
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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.