Golodirsen: First Approval

Heo, Young-A

doi:10.1007/s40265-020-01267-2

Cited by 200 publications

(153 citation statements)

References 4 publications

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“…In December 2019, intravenous golodirsen was approved in the United States to treat DMD patients [63]. In the 168-week phase II study involving 25 patients, all participants showed exon 53 skipping response at week 48, along with an increased dystrophin expression (from 0.095% to 1.109%) [64].…”

Section: Phosphorodiamidate Morpholino Oligomer (Pmo) Modificationmentioning

confidence: 99%

Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update

Sun

Shen

Zhang

et al. 2020

Genes

111

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Neuromuscular disorders encompass a heterogeneous group of conditions that impair the function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being the most common and most severe type of muscular dystrophy, Duchenne muscular dystrophy (DMD), is caused by mutations in the X-linked dystrophin gene. Loss of dystrophin protein leads to recurrent myofiber damage, chronic inflammation, progressive fibrosis, and dysfunction of muscle stem cells. Over the last few years, there has been considerable development of diagnosis and therapeutics for DMD, but current treatments do not cure the disease. Here, we review the current status of DMD pathogenesis and therapy, focusing on mutational spectrum, diagnosis tools, clinical trials, and therapeutic approaches including dystrophin restoration, gene therapy, and myogenic cell transplantation. Furthermore, we present the clinical potential of advanced strategies combining gene editing, cell-based therapy with tissue engineering for the treatment of muscular dystrophy.

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Section: Phosphorodiamidate Morpholino Oligomer (Pmo) Modificationmentioning

confidence: 99%

Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update

Sun

Shen

Zhang

et al. 2020

Genes

111

View full text Add to dashboard Cite

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“…Golodirsen is an antisense oligonucleotide with a single strand of 25 monomers [6]. The subunits are linked through a synthetic neutral phosphorodiamidate morpholino oligomer (PMO) backbone.…”

Section: Golodirsen (Vyondys 53 Tm )mentioning

confidence: 99%

2019 FDA TIDES (Peptides and Oligonucleotides) Harvest

et al. 2020

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2019 has been an excellent year in terms of peptides and oligonucleotides (TIDES) approved by the FDA. Despite the drop in the number of total drugs approved by the FDA in 2019 in comparison with 2018 (48 vs. 59), the total number of TIDES authorized increased (seven vs. three). Year after year, TIDES are increasingly present in therapy, as imaging agents, theragnostic and constituent moieties of other complex drugs, such as antibody drug conjugates. This means a consolidation of these kinds of drugs in the pharmaceutical arena, paving the way in the coming years for the approval of others for diverse medical indications. Here the TIDES approved in 2019 are analyzed in terms of chemical structure, medical target, mode of action, and adverse effects.

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“…Golodirsen is another antisense drug targeted for the treatment of DMD. 57,58 Similarly, as in the case of Exondys51™, it was developed by Sarepta Therapeutics and its mechanism of action is based on the splicing modulation. Golodirsen is a 25-nucleotide phosphorodiamidate morpholino oligomer with the sequence of 5 0 -GTTGCCTCCGGTTCTGAAGGTGTTC-3 0 and triethylene glycol chain incorporated at 5 0 end.…”

Section: Golodirsen (Vyondys 53™)mentioning

confidence: 99%

“…Golodirsen is a 25-nucleotide phosphorodiamidate morpholino oligomer with the sequence of 5 0 -GTTGCCTCCGGTTCTGAAGGTGTTC-3 0 and triethylene glycol chain incorporated at 5 0 end. 58,59 4. In vitro and in vivo studies of antisense oligonucleotides…”

Section: Golodirsen (Vyondys 53™)mentioning

confidence: 99%