baliforsen (ISIS-DMPK) / Ionis - LARVOL DELTA

Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial. (PubMed, Lancet Neurol) - P1/2 | "Baliforsen was generally well tolerated. However, skeletal muscle drug concentrations were below levels predicted to achieve substantial target reduction. These results support the further investigation of ASOs as a therapeutic approach for myotonic dystrophy type 1, but suggest improved drug delivery to muscle is needed."

Journal • P1/2 data • Genetic Disorders • Hematological Disorders • Muscular Dystrophy • Myotonic Dystrophy • Pain • Thrombocytopenia

Degradation of Toxic RNA in Myotonic Dystrophy Using Gapmer Antisense Oligonucleotides. (PubMed, Methods Mol Biol) - "Compared to other antisense oligonucleotides, gapmers maintain the ability to induce RNase H cleavage while having enhanced target binding affinity and nuclease resistance. This chapter will consolidate the different strategies studied thus far to develop a treatment for DM1 through the targeting of toxic repetitive RNA using gapmers."

Journal • Cataract • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy • Ophthalmology