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Genetic Linkage

Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). Available in Italy for three years, Lenmeldy (atidarsagene autotemcel), from Orchard Therapeutics, is groundbreaking, but comes at quite a cost – the $4.25 million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it.


An Ultrarare Neurological Condition


MLD affects the white matter in the brain, causing progressive loss of mobility and sensation, as well as intellectual decline and, ultimately, unresponsiveness.


To continue reading, go to DNA Science, where this post first appeared.

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