GENETIC DISEASE, INBORN
Clinical trials for GENETIC DISEASE, INBORN explained in plain language.
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New drug could help boys with duchenne walk and move longer
Disease control Recruiting nowThis study tests a new medicine called DYNE-251 in boys aged 4 to 18 with Duchenne muscular dystrophy (DMD) who can still walk. The goal is to see if it helps them move better and slows muscle damage. Participants will receive either the drug or a placebo every 4 weeks for about …
Matched conditions: GENETIC DISEASE, INBORN
Phase: PHASE3 • Sponsor: Dyne Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:37 UTC
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Gene-Editing shot aims to halt rare nerve disease in phase 3 trial
Disease control Recruiting nowThis phase 3 trial tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. Participants are randomly assigned to receive either the active treatmen…
Matched conditions: GENETIC DISEASE, INBORN
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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Blood cell harvest could fuel future immune disease cures
Knowledge-focused Recruiting nowThis study collects blood stem cells and immune cells from healthy volunteers and patients with primary immune deficiencies or blood disorders. The cells are used in the lab to develop new gene and cell therapies. Up to 850 adults aged 18-70 will participate. The goal is to advan…
Matched conditions: GENETIC DISEASE, INBORN
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Knowledge-focused
Last updated Jul 02, 2026 00:00 UTC