A toxic gain-of-function variant in MAPK8IP3 provides insights into JIP3 cellular roles
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Positive Clinical Observations from n-Lorem Treated, KIF1A Nano-rare Patient Published in Nature Medicine
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A New Generation of Antisense Oligonucleotides (ASOs) is on the Horizon
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How to think about drug-related toxicities
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The Complexity of Antisense Oligonucleotides (ASOs) for Nano-rare Patients
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