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A Compassionate use study for CER-001 in the Rare Disease LCAT Deficiency or Norum Disease in Europe

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Trial Profile

A Compassionate use study for CER-001 in the Rare Disease LCAT Deficiency or Norum Disease in Europe

Status: Recruiting
Phase of Trial: Clinical Phase Unknown

Latest Information Update: 04 Mar 2025

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At a glance

  • Drugs CER 001 (Primary)
  • Indications Lecithin acyltransferase deficiency
  • Focus Expanded access; Therapeutic Use

    • Most Recent Events

    • 28 Feb 2025 According to an ABIONYX Pharma media release, the company will present the clinical data of CER-001 related to its compassionate use in treated LCAT patients from four European countries, with a view to applying for a marketing authorization (MA) by the end of this year.
    • 14 Jul 2023 New trial record
    • 10 Jul 2023 According to an ABIONYX Pharma media release, the company has been granted a new Compassionate Access Authorization (CAA) in Europe for CER-001 in the rare, untreated kidney disease LCAT Deficiency. Clinical results of the first patients from this study will be communicated exclusively via scientific publication.

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