OSTEOPETROSIS
Clinical trials for OSTEOPETROSIS explained in plain language.
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Gene therapy offers new hope for kids with brittle bone disease
Disease control Recruiting nowThis study tests a new gene therapy for children with a severe inherited bone disease called osteopetrosis. The therapy uses the child's own blood stem cells, modified to fix the genetic defect, and then returned to the body. The goal is to improve blood cell production and survi…
Matched conditions: OSTEOPETROSIS
Phase: PHASE1, PHASE2 • Sponsor: Fondazione Telethon • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Major study tracks rare brain diseases to unlock their secrets
Knowledge-focused Recruiting nowThis 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…
Matched conditions: OSTEOPETROSIS
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:03 UTC