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Updates from EGMEDC and selected coverage from across the genomic medicine community.

4 articles

Screenshot August in wheelchair

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3 March 2026 · Henri van de Vrugt & Laura Roy

August’s Case: When Treatment Exists but Access Does Not

Gene therapy for ultra-rare neurodegenerative diseases is no longer hypothetical, yet access remains uncertain and uneven. The case of August and his parents’ attempts to access experimental genomic medicine for a progressively debilitating condition illustrates how fragmented regulation, funding barriers, and slow clinical translation continue to delay care, often with irreversible consequences.

Recent updates

Earlier announcements and coverage from across the community.

  • Tom Foti is Vice President of Scientific Partnerships at Aldevron webp

    6 May 2026 · Gorm Palmgren

    For Tom Foti, Vice President of Scientific Partnerships at Aldevron, the case with baby KJ at the Children’s Hospital of Philadelphia (CHOP) revealed that rapid genomic medicine relies on relationships and infrastructure established long before a patient reaches crisis.
  • Özcan Met in front of laboratory door

    23 January 2026

    For people with advanced melanoma skin cancer, a treatment utilizing immune cells derived from their own tumours holds great promise. Özcan Met, Associate Professor at Denmark’s National Center for Cancer Immune Therapy (CCIT–DK), is utilizing CRISPR–based gene editing to further improve this personalized, cell-based approach. His work highlights both what it takes to conduct complex clinical trials in an academic setting and why sustaining innovation beyond pilot studies remains challenging.
  • Prof. Martina Cornel, Prof. Rob Wolthuis, Dr Daniël Warmerdam, Department of Human Genetics, Amsterdam UMC.

    27 November 2025 · Henri van de Vrugt

    Personalized gene-editing treatments remain out of reach for most patients in Europe today. To understand why, it helps to look at the front lines. At the Amsterdam UMC ‘CRISPR Clinic’ – one of Europe’s first academic centres dedicated to guiding patients and families on gene-editing options – the systemic gaps become immediately visible.
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