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  • ||||||||||  Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
    [VIRTUAL] Idefirix – A new treatment option (Hall London) -  May 26, 2021 - Abstract #ERAEDTA2021ERA_EDTA_1853;    
    Initial safety and efficacy findings suggest the potential of SRP-9001 therapy for clinically meaningful functional improvements in people with DMD. Sponsored by Hansa Biopharma
  • ||||||||||  Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
    [VIRTUAL] Use of a Novel Bead-based Assay to Measure the Impact of Imlifidase on ABO IgG Antibodies () -  May 18, 2021 - Abstract #ATC2021ATC_2168;    
    ABO-Ab isotype differentiation may be particularly relevant in the context of plasmapheresis, commonly used in ABOi transplant, which more efficiently removes IgM Ab than IgG. HA has limitations in monitoring the effectiveness of imlifidase treatment but this bead-based antibody assessment adequately measures the effect of imlifidase and may provide a tool to clarify if there is a role for imlifidase in ABOi transplantation in individuals with high levels of ABO IgG Ab.
  • ||||||||||  Vyondys 53 (golodirsen) / Sarepta Therap
    Trial completion date, Trial termination, Trial primary completion date:  An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) -  May 17, 2021   
    P4,  N=2, Terminated, 
    HA has limitations in monitoring the effectiveness of imlifidase treatment but this bead-based antibody assessment adequately measures the effect of imlifidase and may provide a tool to clarify if there is a role for imlifidase in ABOi transplantation in individuals with high levels of ABO IgG Ab. Trial completion date: Oct 2023 --> May 2021 | Active, not recruiting --> Terminated | Trial primary completion date: Oct 2023 --> May 2021; The Sponsor has decided to focus their resources on other areas of therapy.
  • ||||||||||  Vyondys 53 (golodirsen) / Sarepta Therap, Translarna (ataluren) / PTC Therap, Exondys 51 (eteplirsen) / Sarepta Therap
    Review, Journal:  Is it the right time for an infant screening for Duchenne muscular dystrophy? (Pubmed Central) -  May 15, 2021   
    Five to eight DMD subjects are believed to be diagnosed. The pilot project would give the opportunity to test in a small population the feasibility of an infant screening programme, which in the near future could be applicable to an entire country.
  • ||||||||||  oprozomib (ONX 0912) / Amgen, SRP-4044 / Sarepta Therap, Ninlaro (ixazomib) / Takeda
    Journal:  Proteasome inhibitors reduce thrombospondin-1 release in human dysferlin-deficient myotubes. (Pubmed Central) -  May 15, 2021   
    Our findings indicate that the ubiquitin-proteasome system might not be the main mechanism of mutant dysferlin degradation. However, its inhibition could help to improve muscle inflammation by reducing TSP-1 release.
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap, Viltepso (viltolarsen) / Nippon Shinyaku
    Clinical, Journal:  Highly sensitive screening of antisense sequences for different types of DMD mutations in patients' urine-derived cells. (Pubmed Central) -  May 15, 2021   
    An essential finding of this study is that the sequence of eteplirsen consistently leads to much lower efficiency than other sequences. These findings underscore the importance of AON sequence optimization by our cellular system, which enables highly sensitive screening of exon skipping drugs that target different types of DMD mutations.
  • ||||||||||  sirolimus / Generic mfg.
    [VIRTUAL] Micro-Dystrophin Gene Therapy Delivery and Therapeutic Plasma Exchange in Non-Human Primates () -  Apr 30, 2021 - Abstract #ASGCT2021ASGCT_758;    
    Methods In Part 1, we intravenously (IV) dosed 5 cohorts (n=3 each) of treatment-naïve NHPs at 2x1014 vg/kg titer (supercoiled qPCR titer method) with rAAVrh74.MHCK7.micro-dystrophin (SRP-9001)...Cohort 5 received rituximab, sirolimus and prednisone (14 days pre, 60 days post)...Conclusion These findings establish TPE as a safe and effective strategy for AAVrh74 vector delivery in NHPs. The results may inform human studies of individuals with preexisting immunity.
  • ||||||||||  SRP-4044 / Sarepta Therap
    [VIRTUAL] Adenine Base Editing to Modulate mRNA Splicing as a Therapeutic Strategy for Duchenne Muscular Dystrophy () -  Apr 30, 2021 - Abstract #ASGCT2021ASGCT_603;    
    In order to test the effect of splice site disruption on exon skipping, we generated a human iPSC line harboring a deletion of dystrophin exon 44...To facilitate in vivo editing, we evaluated both ABE8e and ABE8.17m in AAV-compatible split intein designs. Future studies will evaluate the functional impact this single base pair change can make on the DMD dystrophic phenotype.
  • ||||||||||  Galgt2 gene therapy / Nationwide Children's, Sarepta Therap
    [VIRTUAL] Safety of Delivery of rAAVrh74.MCK.GALGT2 by Isolated Limb Infusion in Two Boys as a Surrogate Gene Therapy for Duchene Muscular Dystrophy () -  Apr 30, 2021 - Abstract #ASGCT2021ASGCT_602;    
    In contrast, subject 2, treated at a younger age and at a higher dose, demonstrated an improvement over 24 months in the NSAA score (from 20 to 23 points), an increase in the 6MW distance (from 405 to 478 m), and only a minimal increase in 100 meter time (45.6 to 48.4 sec), suggesting stability or improvement. These limited data suggest the preliminary safety of delivery of a systemic dose of 1 x 1014 vg/kg, and suggest efficacy at the higher dose in younger patients.
  • ||||||||||  Amondys 45 (casimersen) / Sarepta Therap
    Journal:  Casimersen. (Pubmed Central) -  Apr 22, 2021   
    These data will provide important information for our ongoing clinical development program. No abstract available
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap
    Journal:  Efficient correction of Duchenne muscular dystrophy mutations by SpCas9 and dual gRNAs. (Pubmed Central) -  Apr 20, 2021   
    Based on this predictive repair outcome, efficient in-frame deletion of a part of DMD exon 51 was achieved in HEK293T cells with plasmids expressing SpCas9 and dual gRNAs...Restoration of DMD expression at both the mRNA and protein levels was confirmed in the induced myotubes. With further development, the combination of SpCas9-dual gRNA-corrected DMD patient fibroblasts and transdifferentiation may provide a valuable therapeutic strategy for DMD.
  • ||||||||||  Journal:  Development of Antisense Oligonucleotide Gapmers for the Treatment of Dyslipidemia and Lipodystrophy. (Pubmed Central) -  Mar 18, 2021   
    Volanesorsen, another 20-mer MOE gapmer has shown to be successful in lowering the levels of triglycerides (TGs) in several lipid disorders and has received conditional approval in the European Union for the treatment of Familial chylomicronemia syndrome (FCS) in May 2019 following successful results from phase II/III clinical trials. This chapter focuses on the clinical applications of gapmer AOs for genetic dyslipidemia and lipodystrophy.
  • ||||||||||  Exondys 51 (eteplirsen) / Sarepta Therap
    Clinical, Review, Journal:  Clinical Phenotypes of DMD Exon 51 Skip Equivalent Deletions: A Systematic Review. (Pubmed Central) -  Mar 9, 2021   
    No abstract available This review shows that the majority of exon 51 "skip-equivalent" deletions result in milder (BMD) phenotypes and supports that exon 51 skipping therapy could provide clinical benefit, although we acknowledge that other factors, such as age at treatment initiation or ongoing standard of care, may influence the degree of benefit.
  • ||||||||||  Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
    [VIRTUAL] Micro-dystrophin Gene Therapy Delivery and Therapeutic Plasma Exchange in Non-Human Primates () -  Mar 4, 2021 - Abstract #MDA2021MDA_55;    
    Cohorts 2–4 were treated with prednisone at different time points (Cohort 2: 1 day pre, 30 days post; Cohort 3: 1 day pre, 60 days post; Cohort 4: 14 days pre, 60 days post), and Cohort 5 was treated with rituximab, sirolimus and prednisone (14 days pre, 60 days post)...In the second part of the study, seven of the NHPs dosed in the first part of the study who were positive for AAVrh74 antibodies were treated by TPE prior to a second SRP-9001 dose (prednisone was given 1 day pre, 30 days post)... These findings establish TPE as a safe and efficacious strategy for AAVrh74 vector delivery in NHPs and may inform human studies in individuals with pre-existing immunity, or redosing.
  • ||||||||||  Vyondys 53 (golodirsen) / Sarepta Therap
    Enrollment change, Trial suspension:  An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) -  Mar 3, 2021   
    P4,  N=2, Suspended, 
    These findings establish TPE as a safe and efficacious strategy for AAVrh74 vector delivery in NHPs and may inform human studies in individuals with pre-existing immunity, or redosing. N=12 --> 2 | Recruiting --> Suspended
  • ||||||||||  Journal:  A Neuromuscular-Based Analysis of the Open Payments Program. (Pubmed Central) -  Feb 20, 2021   
    N=12 --> 2 | Recruiting --> Suspended A substantial increase in the annual payments to neuromuscular physicians during the study period is likely due to the development of new treatments, including gene therapy.
  • ||||||||||  Translarna (ataluren) / PTC Therap, Exondys 51 (eteplirsen) / Sarepta Therap
    Review, Journal:  Year in Review 2019: Neuromuscular Diseases. (Pubmed Central) -  Feb 18, 2021   
    Neuromuscular cardiopulmonary medicine is entering a new and exciting phase, with studies that assess the respiratory effect of emerging genetic and molecular therapies. In this year's neuromuscular Year in Review, we focus on Duchenne muscular dystrophy (DMD), reviewing studies that evaluate the respiratory effect of eteplirsen, the cardiopulmonary effects of ataluren, and a study comparing the use of spironolactone to eplerenone for the treatment of DMD-related cardiomyopathy.
  • ||||||||||  Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
    Enrollment open:  An Long-term Follow-up Trial of Kidney Tx Patients Treated With Imlifidase or PE After an AMR (clinicaltrials.gov) -  Feb 4, 2021   
    P=N/A,  N=30, Recruiting, 
    To fully realize the benefits of exon-skipping, including cocktail oligonucleotide-mediated multiple exon-skipping and oligonucleotide drugs for very rare mutations, regulatory challenges need to be addressed in coordination with scientific advances. Not yet recruiting --> Recruiting