# Atamyo Therapeutics Presents Promising Results in the First Patients Treated With Its ATA-200 Gene Therapy in the Clinical Trial Targeting LGMD-R5 Limb-girdle Muscular Dystrophy

_Thursday, July 9, 2026 at 8:00 AM EDT · Science · Latest · Tier 2 — Notable_

![Atamyo Therapeutics Presents Promising Results in the First Patients Treated With Its ATA-200 Gene Therapy in the Clinical Trial Targeting LGMD-R5 Limb-girdle Muscular Dystrophy — Primary](https://static.biospace.com/dims4/default/d32393a/2147483647/strip/true/crop/1120x630+40+0/resize/1440x810!/quality/90/?url=https%3A%2F%2Fk1-prod-biospace.s3.us-east-2.amazonaws.com%2Fbrightspot%2Fcc%2F88%2Fa9fddaf440db946b77e1d45d2e26%2Fbiospace-5.png)

EVRY, France -- Atamyo Therapeutics announced at the MDA Conference 2026 the first safety, pharmacodynamics and efficacy results for its ATA-200 gene therapy in LGMD-2C/R5 limb-girdle muscular dystrophy, the company said. The results are from the first patients treated in a Phase 1b/2 clinical trial conducted at the Powell Gene Therapy Center at the University of Florida by principal investigator Dr. Barry Byrne. Four patients have received the therapy in the ongoing single-center study evaluating safety, pharmacodynamics, efficacy and immunogenicity in children aged 6 to 13 years. Nine-month follow-up data, including muscle biopsies at six months, for the first two patients showed safety was demonstrated and efficacy results were very encouraging, the company said. At a dose of 1.0E+14 vg/kg, more than 90% of muscle fibers expressed the SGCG protein, with 90.2% for patient 1 and 92.1% for patient 2. A significant and sustained reduction in CPK levels and a decrease in transaminases were observed nine months after treatment. Clinical benefits were observed on several parameters in ambulatory patients, particularly in timed functional tests. No serious side effects were observed in the four patients treated. Further results from the ongoing study are expected in the coming months.

## Sources

- [biospace.com](https://www.biospace.com/press-releases/atamyo-therapeutics-presents-promising-results-in-the-first-patients-treated-with-its-ata-200-gene-therapy-in-the-clinical-trial-targeting-lgmd-r5-limb-girdle-muscular-dystrophy)

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