Lyon, France – Austin, Texas, United States – November 19, 2024 – 6 PM CET – Theranexus, a biopharmaceutical
company innovating in the treatment of rare neurological diseases, and the Beyond Batten Disease Foundation
(BBDF) presented the final and positive results of the Phase I/II trial to evaluate Batten-1 in 6 young adult patients
with CLN3 Batten’s disease after 18 months of treatment, at the Child Neurology Society Annual Meeting in San
Diego, 11 to 14th November.
This presentation highlights the strong safety profile of miglustat in CLN3 Batten disease patients and underscores
the positive efficacy data gathered during this trial. The findings demonstrate clear target engagement, a
biological effect, and are suggestive of a stabilization of disease progression over the treatment period. All trial
participants chose to continue receiving miglustat through the BBDF’s early access program, with no safety
concerns reported after two years of ongoing treatment.
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