Atamyo Therapeutics
Clinical trials sponsored by Atamyo Therapeutics, explained in plain language.
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Gene therapy hope for kids with rare muscle-wasting disease
Disease control OngoingThis early-stage trial tests a single intravenous dose of a gene therapy (ATA-200) in 4 children aged 6-12 with limb-girdle muscular dystrophy type 2C/R5 (LGMD2C), a rare genetic muscle-weakening condition. The goal is to see if the treatment is safe and tolerable by delivering a…
Phase: PHASE1 • Sponsor: Atamyo Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Experimental gene therapy targets rare muscle disease
Disease control OngoingThis early-stage trial tests a gene therapy called ATA-100 for people with LGMDR9, a rare genetic muscle disease that causes progressive weakness. Six adults receive a single intravenous infusion of the therapy, which delivers a working copy of the FKRP gene. The main goal is to …
Phase: PHASE1 • Sponsor: Atamyo Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:54 UTC
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New study aims to map rare muscle disease progression
Knowledge-focused TerminatedThis study was designed to track the natural course of gamma-sarcoglycanopathy (LGMDR5), a rare muscle-weakening disease, over two years. Researchers planned to measure changes in muscle strength, walking ability, and daily function in patients aged 6 to 35. The goal was to bette…
Sponsor: Atamyo Therapeutics • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:54 UTC