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 from the last 30 days matching "now recruiting"

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 →
ResearchCLINICALTRIALSMar 27

Trial Now Recruiting: Characterization and Contribution of Genome-wide DNA Methylation (DNA Methylation Episignatures) in Rare Diseases With Prenatal Onset (NCT06475651)

Researchers are looking for 63 participants to help them understand how DNA changes in babies before birth compare to DNA changes after birth. They want to collect DNA from amniotic fluid (the fluid around a baby in the womb) or from tissue samples after delivery to create a reference guide. This guide will help doctors better diagnose rare genetic diseases that start before birth.

WHY IT MATTERSThis trial could improve how doctors diagnose rare genetic diseases in fetuses and newborns by establishing what normal DNA patterns look like before birth, which is currently unknown.
You can act on thisRare Fetal Genetic DiseasesCongenital MalformationRead →

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