Skin pigmentation disorder

This study tests a donor stem cell transplant for people with severe immune system problems, like SCID and Wiskott-Aldrich syndrome. The goal is to help the body make healthy blood cells and fight infections. Participants receive donated stem cells to rebuild their immune system.…

This study tests the long-term safety and effectiveness of pegcetacoplan in 100 people with two rare kidney diseases, C3 glomerulopathy and IC-MPGN. Participants previously completed a one-year trial and saw benefit from the drug. The goal is to see if pegcetacoplan can keep redu…

This study tests a stem cell transplant using a milder chemotherapy-like regimen to treat children and young adults (up to age 28) with various immune system disorders. The goal is to help the donor stem cells settle in the body with fewer side effects. The study involves 20 part…

This program provides early access to pegcetacoplan for people aged 12 and older with C3 glomerulopathy or primary IC-MPGN, rare kidney diseases that can lead to kidney failure. Participants must have active disease with significant protein in their urine. The goal is to offer a …

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study compares two tools, piezotome and periotome, used for gentle tooth extraction before placing a dental implant right away in the front upper teeth. The goal is to see which tool better preserves bone and gum tissue, leading to improved healing and appearance. Thirty adu…

This study collects and analyzes blood, DNA, and tissue samples from 315 adults with pulmonary fibrosis, their relatives, and healthy volunteers. The goal is to learn more about the disease's causes, not to test a new treatment. Researchers hope the findings will guide future the…

This study looks at whether pulse oximeters, which measure oxygen levels, give less accurate readings for newborns with darker skin. Researchers will compare oxygen readings from the device with actual blood oxygen levels in 163 babies of different skin tones. The goal is to find…

This study aims to understand how diet affects the eyes and skin by measuring natural pigments called carotenoids. Researchers will use non-invasive methods to check levels in the eye and skin, and also test blood in some participants. Anyone age 7 or older having a routine eye e…