Non-human animal disease

This study tests a gene therapy called RGX-121 in children aged 4 months to 5 years with Hunter syndrome, a rare genetic disease that affects the brain and body. The therapy delivers a working copy of the missing gene to the central nervous system. Researchers will measure improv…

This study tests a treatment called D-Fi, made from a person's own skin cells that are genetically modified to produce a missing protein. It aims to see if injecting these cells into wounds helps them close completely compared to standard care alone. The trial includes 32 childre…

This early-stage study tests a one-time gene therapy injection into the spinal fluid of children aged 1-18 with CLN7 Batten disease, a rare and fatal genetic brain disorder. The main goal is to check safety, but researchers will also measure changes in movement, thinking, and vis…

This phase 3 trial tests a capsule made from four traditional anti-diabetic herbs (Syzygium cumini, bitter melon, Wrightia tinctoria, and Gymnema sylvestre) in 500 adults with type 2 diabetes. Participants take either the herbal capsule or the standard drug metformin three times …

This study follows 10 people with CLN6 Batten disease who received a single dose of gene therapy (AT-GTX-501) in an earlier trial. Researchers are checking long-term safety and how the disease progresses over time. No new treatment is given in this follow-up.

This early study will test whether weak electrical currents applied to the head can help diagnose balance disorders like vestibular schwannoma and vestibular neuritis. Researchers will measure how 30 people with these conditions sway or move in response to the stimulation. The go…

This early-stage study tests whether ELK-003 eye drops can safely reduce eye symptoms in people with two types of epidermolysis bullosa (EB), a rare condition causing fragile skin and blisters. About 20 participants will first be observed without treatment, then receive the eye d…

This study evaluates a program that aims to improve diets by making healthy foods like fish, chicken, and eggs more available and affordable for low-income households in Mozambique. Researchers will survey over 5,000 men and women to see if the program increases how often they ea…