Newborn screening expands the rare-disease frontier
The expanding newborn-screening panel is changing when and how rare diseases are identified - and what therapy that opens up.
The story rare-disease therapy can tell increasingly depends on identification. Newborn screening expansion - SMA, ALD, others - is what unlocks the early-treatment window many gene therapies need to deliver their best outcomes.
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Published by PatientSpotlight, by PanaceaIntel.