Understanding how adults with childhood genetic diseases plan for their futures

Adults with childhood-onset genetic disease: planning for the future amid therapeutic innovation

['FUNDING_CAREER'] · UNIVERSITY OF PENNSYLVANIA · NIH-11054943

This study is looking at how adults who were diagnosed with genetic diseases as kids are managing their lives and health now that treatments are improving, and it aims to understand their experiences and needs as they grow older.

Quick facts

Phase['FUNDING_CAREER']
Study typeNih_funding
SexAll
SponsorUNIVERSITY OF PENNSYLVANIA (nih funded)
Locations1 site (PHILADELPHIA, UNITED STATES)
Trial IDNIH-11054943 on ClinicalTrials.gov

What this research studies

This research investigates the experiences of adults who were diagnosed with genetic diseases in childhood and how they navigate their futures amid advancements in treatment. It focuses on understanding the psychosocial impacts of living longer than previously expected and how these individuals conceptualize their health and life planning. By employing a disability studies framework, the research aims to gather insights into the evolving prognoses of these patients and their needs as they age. The goal is to ensure that the unique challenges faced by this population are recognized and addressed in the context of genomic medicine.

Who could benefit from this research

Good fit: Ideal candidates for this research are adults who were diagnosed with genetic conditions during childhood and are now navigating adulthood.

Not a fit: Patients who do not have a childhood-onset genetic disease may not receive any benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to improved support and resources for adults with childhood-onset genetic diseases, helping them better plan for their futures.

How similar studies have performed: While there has been some research on childhood genetic conditions, this specific focus on adult experiences and planning is relatively novel and under-explored.

Where this research is happening

PHILADELPHIA, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.