Seminar SeriesPersonalized Splice-modulating Antisense Oligonucleotide Therapy for PEX1-related Zellweger Spectrum Disorder
Seminar SeriesProject Butterfly: from drug development to clinical trial. A personalized antisense oligonucleotide medicine for a rare form of CLN3 Batten Disease
Seminar SeriesPRAX-222 Therapy for a Child: Prenatal Onset SCN2A Developmental and Epileptic Encephalopathy in a Named Patient Setting
Seminar SeriesPotential Treatment: CMT2S Caused By IGHMBP2 Cryptic Splice Site Variant With ASO Based Therapeutic
Seminar SeriesPerspectives on Clinical Outcomes for N=1 Patients: Insights from N1C Clinical Outcomes Workgroup
PublicationsSuppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide
