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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.


