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- || baliforsen (ISIS-DMPK) / Ionis
P1/2 data, Journal: Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial. (Pubmed Central) - Feb 27, 2023
P1/2
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.
- | baliforsen (ISIS-DMPK) / Ionis
Journal: Degradation of Toxic RNA in Myotonic Dystrophy Using Gapmer Antisense Oligonucleotides. (Pubmed Central) - Mar 18, 2021
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.
- | baliforsen (ISIS-DMPK) / Ionis
Trial completion, Enrollment change, Trial primary completion date: A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1 (clinicaltrials.gov) - Nov 3, 2016
P1/2, N=48, Completed, Sponsor: Ionis Pharmaceuticals, Inc.
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. Active, not recruiting --> Completed | N=80 --> 48 | Trial primary completion date: Dec 2016 --> Jul 2016
- ||| baliforsen (ISIS-DMPK) / Ionis
Enrollment closed: A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1 (clinicaltrials.gov) - Sep 20, 2016
P1/2, N=80, Active, not recruiting, Sponsor: Ionis Pharmaceuticals, Inc.
Active, not recruiting --> Completed | N=80 --> 48 | Trial primary completion date: Dec 2016 --> Jul 2016 Recruiting --> Active, not recruiting
- ||| baliforsen (ISIS-DMPK) / Ionis
Enrollment change, Trial primary completion date: A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1 (clinicaltrials.gov) - Jun 20, 2015
P1/2, N=80, Recruiting, Sponsor: Isis Pharmaceuticals
Recruiting --> Active, not recruiting N=36 --> 80 | Trial primary completion date: Mar 2016 --> Dec 2016
- | baliforsen (ISIS-DMPK) / Ionis
Enrollment open: A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1 (clinicaltrials.gov) - Dec 14, 2014
P1/2, N=36, Recruiting, Sponsor: Isis Pharmaceuticals
N=36 --> 80 | Trial primary completion date: Mar 2016 --> Dec 2016 Not yet recruiting --> Recruiting
- | baliforsen (ISIS-DMPK) / Ionis
New P1/2 trial: A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1 (clinicaltrials.gov) - Dec 10, 2014
P1/2, N=36, Not yet recruiting, Sponsor: Isis Pharmaceuticals