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Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer

This is a long-term safety and efficacy study in subjects with CLN6 Batten disease who previously received a single intrathecal administration of AT-GTX-501.

batten
gene therapy
ceroid lipofuscinosis
cln6
neuronal ceroid lipofuscinosis
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  • 16 Feb, 2024
  • 1 location