A bespoke genetic therapy is helping Susannah. Can similar drugs be made at scale for other rare diseases?
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Positive Clinical Observations from n-Lorem Treated, KIF1A Nano-rare Patient Published in Nature Medicine
Case study, published in [...]
Positive Clinical Observations from n-Lorem Treated, KIF1A Nano-rare Patient Published in Nature Medicine
Let’s Discuss Science [...]
Antisense oligonucleotide therapy in an individual with KIF1A-associated neurological disorder
Ziegler, A., Carroll, J., Bain, J.M., Sands, T.T., Fee, R.J., Uher. D., Kanner, C.H., Montes, J., Glass, S., Douville, J., Mignon, L., Gleeson, J.G., Crooke, S.T., Chung, W.K.
Adopting a Rare Son
with Paul Compton
Ion Channel Mutations: The Complexities of Treatment
Arguably, the [...]
Why We Dose Intrathecally
Intrathecal administration [...]
Kuri’s Story: DRPLA
6 minutes
