Resources For Parents and Caregivers
Receiving an ultra-rare diagnosis can place a family in an overwhelming world. We’ve gathered resources for newly diagnosed patients, their parents and/or caregivers.
Navigating the Healthcare System
ClinicalTrials.gov
Many rare and ultra-rare diseases with no FDA-approved treatments do have open clinical trials. These treatments are experimental, and they have been rigorously and scientifically assessed for the potential to improve disease symptoms.
Courageous Parents Network
A rare or ultra-rare diagnosis for your child can be overwhelming. Learn from parents who have been here before, and their tips for how to navigate the healthcare system, advocate for your child, and get the resources they (and you) need.
Ciitizen: managing your patient data and medical notes
Helps collect and manage your child’s medical information. Easily share it with current or future healthcare providers and researchers, as you see fit.
Red Treehouse (by the Ronald McDonald House)
Finding support and information when your child receives a rare disease diagnosis.
Patient Advocacy Umbrella Organizations
Global Genes
When you’re here, you’re part of a globally connected community committed to eliminating the challenges of rare disease. Global Genes is committed to providing information, resources and connections to all communities affected by rare diseases.
EveryLife Foundation
We empower the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments, and cures.
National Organization for Rare Disorders (NORD)
Improving the health and well-being of people with rare diseases by driving advances in care, research, and policy.
Our Inspirations
We are especially grateful to these patient advocacy foundations. Generous with time, advice, and guidance, we follow in their footsteps.
