ResearchCLINICALTRIALSMar 30
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.
Clinical trialCLINICALTRIALSMar 26
Researchers at the National Institutes of Health are looking for up to 4,000 patients with rare metabolic disorders to study and treat. These are conditions where the body cannot properly break down certain substances because of missing or faulty proteins. Patients will receive exams and treatment, mostly as outpatients, though some may need to stay at the NIH Clinical Center for special tests.
WHY IT MATTERSThis trial offers patients with arterial calcification due to CD73 deficiency direct access to specialized NIH researchers and potential treatment options that may not be available elsewhere.