Understanding the palliative care needs of children with rare diseases and their families
Official title: Palliative Care Needs of Children with Rare Diseases and their Families
Funded at Children's Research Institute · NIH-11027250 · Data updated 2026-11-12
This study is all about helping families with children who have rare diseases make tough medical decisions by creating a supportive plan that prepares them for future care options.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Research Institute NIH-funded |
| Lab location | 1 site (Washington, United States) |
| Project start | February 1, 2025 |
| Project end | January 31, 2030 |
| Funding year | 2025 |
| Award (this year) | $790,989 |
| NIH project number | 1R01HD117137-01 |
| Project ID | NIH-11027250 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are families with children aged 0-11 who have been diagnosed with rare diseases.
✕Less likely to help
Patients who do not have rare diseases or are older than 11 years may not receive benefit from this research.
What this research studies
This research focuses on the unique challenges faced by families of children with rare diseases, particularly in making complex medical decisions. It aims to develop and test an advance care planning intervention tailored to these families, helping them prepare for future medical care choices. The study will involve consultations with families and stakeholders to ensure that their basic palliative care needs are addressed before implementing the intervention. By gathering data on these needs, the research seeks to improve support for families navigating difficult healthcare situations.
Why it matters
Potential benefit: If successful, this research could provide families with better tools and support for making informed medical decisions for their children with rare diseases.
How similar studies have gone: While there is limited research specifically addressing the palliative care needs of children with rare diseases, similar approaches in pediatric advance care planning have shown promise in other contexts.
Where this research is happening
-
Children's Research Institute
Washington, United States Active
Researchers
- Principal investigator: Lyon, Maureen Ellen — Children's Research Institute
- Study coordinator: Lyon, Maureen Ellen
Related research
Other NIH-funded projects on the same topics.
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Palliative care and advance planning for children with rare diseases and their families
This project offers a family-centered advance care planning program to help parents of young children with rare diseases address palliative care needs and make future medical decisions.
-
Liver pathobiology from rare liver diseases
A study at UCSF looks at why rare liver conditions cause liver damage, with the aim of finding new ways to diagnose and treat chronic liver disease.
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Data Management and Coordinating Center support for Rare Diseases Research
A national rare diseases research program is funding a center to manage data and coordinate studies to help improve diagnosis, care, and potential treatments for children with rare conditions.
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EEG-based drug repurposing for rare CNS diseases
A new approach uses EEG brain signatures to find existing drugs that might help people with rare nervous-system disorders.
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Ethics of Engaging Patient Groups in Drug Development for Rare Diseases
This grant asks how patient and family groups can ethically help shape rare-disease drug development from idea to market, with input from patients, families, doctors, and researchers.
-
Center for Polyamine Disorders group study of rare metabolic disorders
A research consortium is studying a group of rare metabolic disorders linked to polyamines and aims to understand symptoms, biomarkers, and disease progression to improve care.
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.