Cure My Disease
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Clinical trials sponsored by Cure Rare Disease, Inc, explained in plain language.
Researchers are testing an experimental gene-editing therapy called CRD-TMH-001 in one person with a rare mutation of Duchenne muscular dystrophy. The participant receives a single intravenous dose. The study's main goal is to measure safety and tolerability by tracking serious a…
Phase 1 • Sponsor: Cure Rare Disease, Inc • Aim: Cure
Last updated Oct 21, 2026 18:00 UTC