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A single-patient, investigator-initiated study of RGX-181 for patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease

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Trial Profile

A single-patient, investigator-initiated study of RGX-181 for patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 23 Dec 2022

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At a glance

  • Drugs Pioglitazone deuterated (Primary)
  • Indications Neuronal ceroid lipofuscinosis
  • Focus Adverse reactions; First in man

    • Most Recent Events

    • 23 Dec 2022 New trial record
    • 21 Dec 2022 According to a REGENXBIO media release, Carolina Fischinger, MD, PhD, is the principle investigator in the trial.
    • 21 Dec 2022 According to a REGENXBIO media release, physician investigators at the Hospital de Clinicas in Porto Alegre, Brazil have dosed the first child with CLN2 disease with RGX-181 in the study.

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