Eteplirsen for the treatment of Duchenne muscular dystrophy

@article{Mendell2013EteplirsenFT,
  title={Eteplirsen for the treatment of Duchenne muscular dystrophy},
  author={Jerry R. Mendell and Louise R. Rodino‐Klapac and Zarife Sahenk and Kandice Roush and Loren Bird and Linda P. Lowes and Lindsay N Alfano and A. M. Gomez and Sarah Lewis and Janaiah Kota and Vinod Malik and Kim Shontz and Christopher M. Walker and Kevin M. Flanigan and Marco Corridore and John R. Kean and Hugh D. Allen and Christopher J Shilling and Kathleen R Melia and Peter Sazani and Jay B. Saoud and Edward M. Kaye},
  journal={Annals of Neurology},
  year={2013},
  volume={74}
}
In prior open‐label studies, eteplirsen, a phosphorodiamidate morpholino oligomer, enabled dystrophin production in Duchenne muscular dystrophy (DMD) with genetic mutations amenable to skipping exon 51. The present study used a double‐blind placebo‐controlled protocol to test eteplirsen's ability to induce dystrophin production and improve distance walked on the 6‐minute walk test (6MWT). 
Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study
The novel morpholino antisense oligonucleotide viltolarsen targets exon 53 of the dystrophin gene, and could be an effective treatment for patients with Duchenne muscular dystrophy (DMD). We
Eteplirsen therapy for Duchenne muscular dystrophy: skipping to the front of the line
TLDR
FDA approval of the exon-skipping drug eteplirsen is provisional, pending results of an ongoing phase III clinical trial, and came after much debate.
Eteplirsen Approved for Duchenne Muscular Dystrophy: The FDA Faces a Difficult Choice.
  • C. Stein
  • Biology, Medicine
    Molecular therapy : the journal of the American Society of Gene Therapy
  • 2016
TLDR
Eeplirsen is a 30-mer phosphomorpholidate oligonucleotide that has been evaluated in young male patients with the devastating disease known as Duchenne muscular dystrophy, characterized by mutations in the dystrophin gene that take the messenger RNA reading frame out of frame.
Duchenne and Becker muscular dystrophies.
Early phase 2 trial of TAS‐205 in patients with Duchenne muscular dystrophy
TLDR
This randomized, double‐blind, placebo‐controlled early phase 2 study aimed to assess the efficacy and safety of the novel selective HPGDS inhibitor, TAS‐205, with exploratory measures in male DMD patients aged ≥5 years.
Exon skipping therapy for Duchenne muscular dystrophy.
Current and Emerging Therapies for Duchenne Muscular Dystrophy
  • M. Crone, J. Mah
  • Medicine, Biology
    Current Treatment Options in Neurology
  • 2018
TLDR
There are a growing number of therapeutic options to treat males with DMD, and treatment of the underlying pathobiology, such as micro-dystrophin gene replacement, exon skipping, stop codon read-through agents, and utrophin modulators showed variable success.
What Is in the Myopathy Literature?
We review the development of exon 51 skipping therapy with eteplirsen for Duchenne muscular dystrophy, including the recent report of long-term, sustained dystrophin production. Studies of the
...
1
2
3
4
5
...

References

SHOWING 1-10 OF 42 REFERENCES
Efficacy of systemic morpholino exon‐skipping in duchenne dystrophy dogs
TLDR
This work sought to test efficacy and toxicity of intravenous oligonucleotide (morpholino)‐induced exon skipping in the DMD dog model and found it safe and effective.
Dystrophin immunity in Duchenne's muscular dystrophy.
TLDR
The potential for T-cell immunity to self and nonself dystrophin epitopes should be considered in designing and monitoring experimental therapies for Duchenne's muscular dystrophy.
Therapeutics in Duchenne muscular dystrophy
Evidence‐based path to newborn screening for duchenne muscular dystrophy
TLDR
A 2‐tier system using the dried blood spot to first assess CK with follow‐up DMD gene testing is introduced to assess CK in Duchenne muscular dystrophy.
Local dystrophin restoration with antisense oligonucleotide PRO051.
TLDR
Intramuscular injection of antisense oligonucleotide PRO051 induced dystrophin synthesis in four patients with Duchenne's muscular dystrophy who had suitable mutations, suggesting that further studies might be feasible.
Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense.
TLDR
The application of multiexon skipping may provide a more uniform methodology for a larger group of patients with DMD, and would be therapeutic for a series of patients carrying different DMD-causing mutations.
Systemic administration of PRO051 in Duchenne's muscular dystrophy.
TLDR
Systemically administered PRO051 showed dose-dependent molecular efficacy in patients with Duchenne's muscular dystrophy, with a modest improvement in the 6-minute walk test after 12 weeks of extended treatment.
...
1
2
3
4
5
...