Regulatory GuidancesClinical Recommendations Draft GuidanceIND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases:...
PublicationsAntisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic later
PublicationsSuppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide
