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viltolarsen

antisense oliogonucleotide that targets exon 53 in the dystrophin gene

Also Known As:

viltepso

Networked: 17 relevant articles (2 outcomes, 4 trials/studies)

Relationship Network

Bio-Agent Context: Research Results

Nucleic Acids, Nucleotides, and Nucleosides: 1
- Nucleotides: 15763
  - Polynucleotides: 218
    - Oligonucleotides: 5145
      - viltolarsen: 17

Experts

1.	CINRG DNHS Investigators: 2 articles (01/2022 - 01/2020)
2.	Clemens, Paula R: 2 articles (01/2022 - 01/2020)
3.	Connolly, Anne M: 2 articles (01/2022 - 01/2020)
4.	Harper, Amy D: 2 articles (01/2022 - 01/2020)
5.	Hoffman, Eric P: 2 articles (01/2022 - 01/2020)
6.	Mah, Jean K: 2 articles (01/2022 - 01/2020)
7.	McDonald, Craig M: 2 articles (01/2022 - 01/2020)
8.	Rao, Vamshi K: 2 articles (01/2022 - 01/2020)
9.	Smith, Edward C: 2 articles (01/2022 - 01/2020)
10.	Zaidman, Craig M: 2 articles (01/2022 - 01/2020)

Related Diseases

1.	Duchenne Muscular Dystrophy (Muscular Dystrophy, Becker) 01/01/2020 - "Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial." 01/01/2023 - "Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy." 01/01/2023 - "Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy." 01/01/2023 - "Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study." 12/01/2020 - "Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study."
2.	Inborn Genetic Diseases (Disease, Hereditary) 01/01/2023 - "Viltolarsen is a phosphorodiamidate morpholino antisense oligonucleotide (PMO) designed to skip exon 53 of the DMD gene for the treatment of Duchenne muscular dystrophy (DMD), one of the most common lethal genetic disorders characterized by progressive degeneration of skeletal muscles and cardiomyopathy. "
3.	Disease Progression 01/01/2023 - "For the primary efficacy outcome (TTSTAND), viltolarsen-treated patients showed stabilization of motor function over the first two years and significant slowing of disease progression over the following two years compared with the CINRG DNHS control group which declined. "
4.	Cardiomyopathies (Cardiomyopathy) 01/01/2023 - "Viltolarsen is a phosphorodiamidate morpholino antisense oligonucleotide (PMO) designed to skip exon 53 of the DMD gene for the treatment of Duchenne muscular dystrophy (DMD), one of the most common lethal genetic disorders characterized by progressive degeneration of skeletal muscles and cardiomyopathy. "
5.	Muscular Diseases (Myopathy) 05/01/2023 - "Specific therapy for genetic myopathy is still limited to enzyme replacement therapy for Pompe disease with alglucosidase alfa and avalglucosidase alfa, and exon skipping therapy with antisense nucleotide viltolarsen for about 7% of patients with Duchenne muscular dystrophy. "

Related Drugs and Biologics

1.	Antisense Oligonucleotides
2.	Morpholinos
3.	Dystrophin
4.	casimersen
5.	golodirsen
6.	eteplirsen
7.	viltolarsen
8.	RNA (Ribonucleic Acid)
9.	Nucleotides
10.	2019-nCoV Vaccine mRNA-1273

Related Therapies and Procedures

1.	Therapeutics
2.	Enzyme Replacement Therapy