Pelizaeus-Merzbacher spectrum disorder

This study tests whether adding low-dose radiation to the drug elranatamab can better kill cancer cells in people with multiple myeloma that has come back or not responded to treatment and has spread outside the bone. About 34 adults will receive the combination. The goal is to s…

This early-stage study tests a new medicine called ION356 in 24 boys aged 2 to 17 with Pelizaeus-Merzbacher disease, a rare genetic brain disorder. The main goal is to check if the drug is safe and how the body processes it. The drug is given through a spinal injection to target …

This early-stage trial tests whether adding special cells (DUOC-01) into the spinal fluid is safe for children with inherited metabolic diseases that damage the brain. Participants are ages 1 week to 21 years and are already receiving a standard umbilical cord blood transplant. T…

This study compares two surgeries for stress urinary incontinence (leaking urine when coughing or sneezing) in women. One method uses a synthetic mesh sling, while the other repairs natural tissues without mesh. Researchers will track symptom improvement, recovery time, pain, and…

This observational study is following 32 boys aged 6 months to 17 years with Pelizaeus-Merzbacher disease (PMD), a rare genetic disorder affecting the brain's white matter. Researchers are measuring biomarkers in spinal fluid and blood, brain scans, and motor and cognitive skills…

This study follows 1500 people with rare genetic brain disorders to learn how these diseases progress. Researchers measure thinking, movement, and daily living skills over time, and also look at brain scans and body fluids. The goal is to better understand the diseases and how tr…

This study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…