n-Lorem Foundation (free individualized ASO medicines)
n-Lorem (Ionis-founded)
Discovers, develops, and supplies individualised antisense oligonucleotide medicines free for life to 'nano-rare' patients — roughly 1–30 patients worldwide sharing a mutation. 240+ patients have been accepted to date.
BTHS overall exceeds nano-rare scale, but n-Lorem counts by specific mutation, so a private TAFAZZIN variant could qualify. ASOs cannot replace lost gene function, so most loss-of-function TAFAZZIN mutations are unsuitable; splice-correction or upregulation strategies for particular variants remain possible through physician inquiry.
Applications come from research physicians at tertiary centers experienced with INDs, for fully genomically characterized patients.
Rolling, via the physician application portal.
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Last updated August 28, 2026 · Suggest an update to this entry