Training future researchers in rare genetic disorders affecting the brain
Official title: Predoctoral Preparatory Program (P3) in Translational Research
Funded at Children's Hosp of Philadelphia · NIH-11077757 · Data updated 2026-11-12
This program is all about teaching and training new research coordinators to help find better treatments for rare genetic disorders called leukodystrophies, which affect how the brain develops, so they can make a real difference in this important field.
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 start | May 1, 2024 |
| Project end | April 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $98,855 |
| NIH project number | 5R25TR005269-02 |
| Project ID | NIH-11077757 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are young scholars or early-career professionals interested in pursuing a career in rare disease research.
✕Less likely to help
Patients who are not interested in research careers or who are already established in their research paths may not benefit from this program.
What this research studies
This program aims to educate and train junior research coordinators in the field of rare genetic disorders known as leukodystrophies, which impact brain myelin development. By providing formal education and hands-on experience, the initiative seeks to inspire a new generation of researchers dedicated to advancing treatments for these conditions. Participants will engage with a network of leading academic institutions and gain exposure to translational research, enhancing their career prospects in rare disease research. The program is designed to foster long-term commitment to this critical area of medicine.
Why it matters
Potential benefit: If successful, this research could lead to a stronger pipeline of researchers focused on developing therapies for rare genetic disorders, ultimately improving patient outcomes.
How similar studies have gone: Other initiatives have successfully trained researchers in specialized fields, indicating that this approach has potential for success.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Adang, Laura Ann — Children's Hosp of Philadelphia
- Study coordinator: Adang, Laura Ann
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.