Rare disease news

FDA approvals, research breakthroughs, clinical trials, and advocacy updates

Curated and summarized by AI for patients and caregivers

🔍
AllDrug approvalsClinical trialsResearchGrants & fundingAdvocacy & policyPipeline
Show:All newsBreaking onlyImportant & breaking
Date:7 days30 days90 daysAll time

2 articles matching "undiagnosed diseases"

ResearchCLINICALTRIALSMar 30

Trial Now Recruiting: Caregiving Networks Across Disease Context and the Life Course (NCT05007990)

This study is looking for 2,800 people to help researchers understand how being a caregiver affects a person's health and stress levels over time. The study includes caregivers of people with rare diseases like Batten disease, Tay-Sachs disease, and other inherited metabolic disorders, as well as caregivers of people with undiagnosed diseases. By learning more about caregiver stress, researchers hope to find ways to better support the millions of unpaid family caregivers in the U.S.

WHY IT MATTERSIf you or a family member care for someone with a rare inherited metabolic disorder, Batten disease, Tay-Sachs, or an undiagnosed disease, this study directly addresses the health challenges caregivers face and could lead to better support resources.
You can act on thisBatten diseaseTay-Sachs diseaseInherited metabolic disordersRead →
Clinical trialCLINICALTRIALSMar 26

Trial Now Recruiting: Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network (NCT02450851)

Researchers are looking for 20,000 people with rare diseases that doctors haven't been able to diagnose yet. Many patients spend years going to different doctors and getting repeated tests without finding answers. This study, run by the National Human Genome Research Institute, aims to help solve medical mysteries by using genetic testing and careful evaluation to figure out what's causing people's symptoms.

WHY IT MATTERSIf you or a family member has spent years seeking a diagnosis without answers, this large national study offers free genetic evaluation and expert medical review specifically designed to identify undiagnosed rare diseases.
You can act on thisundiagnosed genetic disordersrare genetic diseasesRead →

Get personalized rare disease news

Follow your conditions to see news about the diseases that matter to you — FDA approvals, trial openings, and research breakthroughs.

Create free account →Browse diseases