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  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap, Spinraza (nusinersen) / Biogen, Ionis
    Review, Journal:  Genetic therapies for inherited neuromuscular disorders. (Pubmed Central) -  Oct 18, 2019   
    Such translational research has led to the approval of two genetic therapies by the US Food and Drug Administration: eteplirsen for Duchenne muscular dystrophy and nusinersen for spinal muscular atrophy, which are both antisense oligonucleotides that modify pre-mRNA splicing. In this Review we aim to discuss new genetic therapies and ongoing clinical trials for Duchenne muscular dystrophy, spinal muscular atrophy, and other less common childhood neuromuscular disorders.
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap
    Journal:  Why dystrophin quantification is key in the eteplirsen saga. (Pubmed Central) -  Aug 30, 2019   
    In this Review we aim to discuss new genetic therapies and ongoing clinical trials for Duchenne muscular dystrophy, spinal muscular atrophy, and other less common childhood neuromuscular disorders. No abstract available
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap
    Journal:  Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. (Pubmed Central) -  Aug 15, 2019   
    N=24 --> 40 | Trial completion date: Dec 2021 --> Jul 2022 | Trial primary completion date: Dec 2021 --> Jul 2022 Taken together, the 4 assays, each based on unique evaluation mechanisms, provided evidence of eteplirsen muscle cell penetration, exon skipping, and induction of novel dystrophin expression.
  • ||||||||||  Translarna (ataluren) / PTC Therapeutics, Sanofi, Exondys 51 (eteplirsen) / Sarepta Therap
    Journal:  A new era in the management of Duchenne muscular dystrophy. (Pubmed Central) -  Jul 26, 2019   
    Exon 51 skipping with eteplirsen demonstrated a structural and functional effect in a small group of patients and received restricted approval in the USA...Scientific, economic, and political efforts are needed to make effective therapies available more quickly. Effective therapies should be made available more quickly to patients in low-income regions.
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap
    Clinical, Journal:  Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. (Pubmed Central) -  Jul 23, 2019   
    They maintained cardiac and upper limb function through combined week 240, with outcomes similar to those of the patients who remained ambulatory. Dystrophin production was confirmed following eteplirsen treatment.Despite the loss of ambulation, other markers of disease progression remained relatively stable in the eteplirsen-treated twin patients and were similar to those of the ambulatory patients.
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap
    Trial completion:  Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy (clinicaltrials.gov) -  Jul 10, 2019   
    P2,  N=12, Completed, 
    Dystrophin production was confirmed following eteplirsen treatment.Despite the loss of ambulation, other markers of disease progression remained relatively stable in the eteplirsen-treated twin patients and were similar to those of the ambulatory patients. Active, not recruiting --> Completed
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap
    Trial completion:  PROMOVI: Study of Eteplirsen in DMD Patients (clinicaltrials.gov) -  Jun 28, 2019   
    P3,  N=109, Completed, 
    Active, not recruiting --> Completed Active, not recruiting --> Completed
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap
    Journal:  What Is in the Myopathy Literature? (Pubmed Central) -  Jun 15, 2019   
    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...Regarding the category of autoimmune myopathies, we discuss an article on the clinical and laboratory features associated with PM/Scl antibodies in comparison with other autoimmune myopathy subgroups. Finally, the overall increase in mortality in inflammatory myopathies is highlighted in a recent report from Sweden.
  • ||||||||||  Vyondys 53 (golodirsen) / Sarepta Therap
    Trial completion:  Phase I/II Study of SRP-4053 in DMD Patients (clinicaltrials.gov) -  Jun 13, 2019   
    P1/2,  N=39, Completed, 
    Finally, the overall increase in mortality in inflammatory myopathies is highlighted in a recent report from Sweden. Active, not recruiting --> Completed
  • ||||||||||  Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
    Enrollment open:  A Study of Imlifidase in Patients With Guillain-Barré Syndrome (clinicaltrials.gov) -  Jun 12, 2019   
    P2,  N=30, Recruiting, 
    Active, not recruiting --> Completed Not yet recruiting --> Recruiting
  • ||||||||||  Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
    Enrollment change, Post-transplantation:  Ides in Highly Sensitized (HS) Patients Awaiting Kidney Transplantation (clinicaltrials.gov) -  Jun 7, 2019   
    P1/2,  N=25, Completed, 
    N=12 --> 4 | Trial completion date: Jan 2021 --> Apr 2021 | Trial primary completion date: Jan 2021 --> Apr 2021 N=17 --> 25
  • ||||||||||  Review, Journal:  Genetic neuromuscular disorders: living the era of a therapeutic revolution. Part 2: diseases of motor neuron and skeletal muscle. (Pubmed Central) -  May 26, 2019   
    Exon skipping technology was and is currently tested in many phase 3 trials, and eteplirsen received a conditional approval by FDA for patients amenable to exon 51 skipping, but not by EMA...Other innovative approaches are under investigation, i.e., gene therapy in X-linked myotubular myopathy and Pompe disease, and antisense oligonucleotides in myotonic dystrophy type 1. Positive evidences are discussed about lamotrigine and ranolazine in non-dystrophic myotonias, chaperons in Pompe disease, and nucleosides in mitochondrial DNA depletion induced by thymidine kinase 2 deficiency.
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap, Spinraza (nusinersen) / Biogen, Ionis
    Journal:  The therapeutic potential of RNA regulation in neurological disorders. (Pubmed Central) -  Apr 18, 2019   
    Expert opinion: Targeting gene expression at the RNA level is a promising strategy to treat genetic neurological disorders. Safe administration, long-term efficacy and potential side effects, however, still need careful evaluation before RNA therapeutics can be applied on a larger scale.
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap, drisapersen (GSK2402968) / GSK
    Retrospective data, Review, Journal:  Exon skipping for Duchenne muscular dystrophy: a systematic review and meta-analysis. (Pubmed Central) -  Apr 17, 2019   
    The small number of RCTs with relatively small numbers of participants indicate the difficulty in conducting sufficiently powered studies of DMD. Prospectively planned meta-analysis and utilization of the real-world data may provide a more precise estimate of the effect of exon skipping in this disease.
  • ||||||||||  Journal:  Invention and Early History of Exon Skipping and Splice Modulation. (Pubmed Central) -  Apr 3, 2019   
    ...In fact, two splice-switching AOs have recently obtained approval from the US Food and Drug Administration: eteplirsen (Exondys 51) for Duchenne muscular dystrophy, and nusinersen (Spinraza) for spinal muscular atrophy...In this chapter, we discuss the early development of AO-based splice modulation therapy-its invention, first applications, and its evolution into the approach we are now familiar with. We give a more extensive history of exon skipping in particular, as it is the splice modulation approach given the most focus in this book.
  • ||||||||||  Clinical, Journal:  An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases. (Pubmed Central) -  Apr 3, 2019   
    ...Antisense-mediated exon skipping is most extensively studied in Duchenne muscular dystrophy (DMD) and has developed from in vitro proof-of-concept studies to clinical trials targeting various single exons such as exon 45 (casimersen), exon 53 (NS-065/NCNP-01, golodirsen), and exon 51 (eteplirsen)...In hopes of achieving clinical success parallel to DMD, exon skipping and splice modulation are also being studied in other muscular dystrophies, such as Fukuyama congenital muscular dystrophy (FCMD), dysferlinopathy including limb-girdle muscular dystrophy type 2B (LGMD2B), Miyoshi myopathy (MM), and distal anterior compartment myopathy (DMAT), myotonic dystrophy, and merosin-deficient congenital muscular dystrophy type 1A (MDC1A). This chapter also summarizes the development of antisense-mediated exon skipping therapy in diseases such as Usher syndrome, dystrophic epidermolysis bullosa, fibrodysplasia ossificans progressiva (FOP), and allergic diseases.
  • ||||||||||  Journal:  Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion. (Pubmed Central) -  Apr 3, 2019   
    ...The approval of Exondys 51 (eteplirsen) and Spinraza (nusinersen) for the treatment of patients with Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) was the most noteworthy accomplishment in 2016...The mechanism of mRNA splicing is highly complex, and the efficacy of AONs is often unpredictable. We will discuss the design of effective AONs for exon skipping and exon inclusion in this chapter.
  • ||||||||||  golodirsen (SRP-4053) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap, viltolarsen (NS-065) / Nippon Shinyaku
    Preclinical, Journal:  Quantitative Evaluation of Exon Skipping in Immortalized Muscle Cells In Vitro. (Pubmed Central) -  Apr 3, 2019   
    ...This is the case with eteplirsen, an exon 51-skipping AO that is the first and only FDA-approved drug for DMD to date...Aside from allowing for the quantitative evaluation of candidate AOs based on their exon skipping efficiency and dystrophin protein rescue levels, these immortalized cells are stable, pure, easy to grow, and not subject to confounding by senescence-related issues. This procedure enables a more reliable screening of AOs prior to their entry in clinical trials and greatly facilitates the search for more efficacious candidate exon skipping AOs for DMD treatment.
  • ||||||||||  CRISPR-Cas9 gene editing therapeutic / CRISPR Therap
    Clinical, Journal:  Creation of DMD Muscle Cell Model Using CRISPR-Cas9 Genome Editing to Test the Efficacy of Antisense-Mediated Exon Skipping. (Pubmed Central) -  Apr 3, 2019   
    ...The approval of Exondys 51 (eteplirsen) targeting exon 51 was the most noteworthy accomplishment in 2016...Furthermore, many DMD mutations are very rare and it is hard to find a patient with a specific mutation for muscle biopsy in many cases. Here, we describe a novel approach to create an immortalized muscle cell line with a DMD deletion mutation using the human rhabdomyosarcoma (RD) cell line and the CRISPR/Cas9 system that can be used to test the efficacy of exon skipping.
  • ||||||||||  golodirsen (SRP-4053) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap, viltolarsen (NS-065) / Nippon Shinyaku
    Preclinical, Journal:  Systemic Injection of Peptide-PMOs into Humanized DMD Mice and Evaluation by RT-PCR and ELISA. (Pubmed Central) -  Apr 3, 2019   
    Researchers have previously relied on high-performance liquid chromatography (HPLC) or liquid chromatography-mass spectrometry (LC/MS) methods for detecting PPMO uptake, but an enzyme-linked immunosorbent assay (ELISA) has been shown to have greater sensitivity. Here, we present methodologies to determine the uptake efficiency of a PPMO into the heart and efficacy of exon 51 skipping by a PPMO injected retro-orbitally into a humanized DMD mouse model via ELISA and RT-PCR, respectively.
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap
    Preclinical, Journal:  In Vivo Evaluation of Single-Exon and Multiexon Skipping in mdx52 Mice. (Pubmed Central) -  Apr 3, 2019   
    Multi-exon skipping utilizing ASOs can theoretically treat 80-90% of patients with DMD. Here, we describe the systemic delivery of a cocktail of ASOs to skip exon 51 and exons 45-55 in the mdx52 mouse, an exon 52 deletion model of DMD produced by gene targeting, and the evaluation of their efficacies in vivo.
  • ||||||||||  golodirsen (SRP-4053) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap, viltolarsen (NS-065) / Nippon Shinyaku
    Preclinical, Journal:  In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs. (Pubmed Central) -  Apr 3, 2019   
    ...The FDA conditionally approved the first exon-skipping AON, called eteplirsen (brand name ExonDys51), targeting exon 51 of the DMD gene, in late 2016...Although the success of multiple exon skipping in a DMD dog model has made a significant impact on the development of therapeutics for DMD, unmodified AONs such as phosphorodiamidate morpholino oligomers (PMOs) have little efficacy in cardiac muscles. Here, we describe our technique of intravenous injection of a cocktail of peptide-conjugated PMOs (PPMOs) to skip multiple exons, exons 6 and 8, in both skeletal and cardiac muscles in dystrophic dogs and the evaluation of the efficacy and toxicity.
  • ||||||||||  sirolimus / generics
    Journal:  Morpholino-Mediated Exon Skipping Targeting Human ACVR1/ALK2 for Fibrodysplasia Ossificans Progressiva. (Pubmed Central) -  Apr 3, 2019   
    Here, we describe a method to reduce ACVR1 expression in FOP patient cells by exon skipping in ACVR1 mRNAs using phosphorodiamidate morpholino oligomers (PMOs). This strategy can be applied to the screen to select antisense oligomers to knockdown not only ACVR1 but also genes which cause other autosomal-dominant genetic diseases.
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap, viltolarsen (NS-065) / Nippon Shinyaku
    Journal:  Exon Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy. (Pubmed Central) -  Apr 3, 2019   
    These findings support the theory that PMO entry into fibers is dependent on the developmental stage in myogenesis rather than on dystrophinless muscle membranes, and provide a platform for the future development of PMO-mediated therapies for a variety of muscular disorders, such as MDC1A, that involve active muscle regeneration. Herein, we describe the methods for PMO transfection/injection and evaluation of the efficacy of exon skipping in the laminin-α2-deficient dy /dy mouse model both in vitro and in vivo.