"Eteplirsen"

Deflazacort, Eteplirsen, and Golodirsen for Duchenne Muscular Dystrophy: Effectiveness and Value ©Institute for Clinical and Economic Review, 2019 Deflazacort, Eteplirsen, and Golodirsen for Duchenne Muscular Dystrophy: Effectiveness and Value Final Evidence Report August 15, 2019 Prepared for ©Institute for Clinical and Economic Review, 2019 Page ii Final Evidence Report – Deflazacort, Eteplirsen, and Golodirsen for DMD ICER Staff and Consultants University of Illinois at Chicago College of Pharmacy Modeling Group Grace A. Lin, MD Associate Professor of Medicine and Health Policy University of California, San Francisco Foluso Agboola, MBBS, MPH Director, Evidence Synthesis ICER Noemi Fluetsch, MPH Research Assistant, Health Economics and Outcomes Research ICER Varun M

A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment. Studies 4658-201/202 (201/202) evaluated treatment effects of eteplirsen over 4 years in patients with Duchenne muscular dystrophy and confirmed exon-51 amenable genetic mutations. Chart review Study 4658-405 (405 ) further followed these patients while receiving eteplirsen during usual clinical care. To compare long-term clinical outcomes of eteplirsen-treated patients from Studies 201/202/405 with those of external controls. Median total follow-up time was approximately 6 years of eteplirsen treatment. Outcomes included loss of ambulation (LOA) and percent-predicted forced vital capacity (FVC%p). Time to LOA

Delays in pulmonary decline in eteplirsen-treated patients with Duchenne muscular dystrophy. Pulmonary decline is a major issue in patients with Duchenne muscular dystrophy (DMD). Eteplirsen is a United States-approved treatment for patients with DMD and exon 51 skip-amenable mutations. Previous analyses have shown that eteplirsen is associated with a statistically significant attenuation of pulmonary decline. In this study we evaluate the effect of eteplirsen treatment from newly available data sources on pulmonary function over time in patients with DMD. We used a post hoc pooled analysis to compare the percentage of predicted forced vital capacity (FVC%p) and projected time with pulmonary function milestones in patients with DMD and exon 51 skip-amenable mutations receiving eteplirsen

Eteplirsen for Duchenne muscular dystrophy in patients amenable to exon 51 skipping Eteplirsen for Duchenne muscular dystrophy in patients amenable to exon 51 skipping ..

Sarepta, eteplirsen: anecdote, data, surrogate outcomes, and the FDA Sarepta, eteplirsen: anecdote, data, surrogate outcomes, and the FDA – Bad Science Bad ScienceSearch * TED TalkCollected JournalismThis Nerdy BookThis Great BookT-shirtsCategories * ABPI (3) * academic pr (4) * academic publishing (6) * adverts (45) * africa (28) * aids (6) * alltrials campaign (16) * alternative medicine (190 ) * survey data (5) * susan greenfield (1) * systematic reviews (10) * tamiflu (1) * teaching resources (2) * tobacco (1) * toys (1) * trial registers (6) * uncertainty (4) * utter nonsense (3) * vaccines (2) * very basic science (52) * video (3) * water (18) * weight loss (10) * whistleblowers (1)Sarepta, eteplirsen: anecdote, data, surrogate outcomes, and the FDASeptember 30th, 2016 by Ben Goldacre

Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. This analysis aims to describe the outcomes of two nonambulatory patients with Duchenne muscular dystrophy (DMD) who participated in two clinical studies. The two consecutive trials of eteplirsen (studies 201 and 202) were conducted in patients with DMD (N = 12) and confirmed genetic mutations amenable to exon 51 skipping.In study 201, 12 patients were randomized to receive once-weekly, double-blind intravenous infusions of eteplirsen 30 or 50 mg/kg or placebo for 24 weeks; patients then received open-label eteplirsen during weeks 25 through 28. All 12 patients continued onto open-label extension study 202 and received long-term treatment with eteplirsen. We compared cardiac, pulmonary, and upper limb

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. To describe the quantification of novel dystrophin production in patients with Duchenne muscular dystrophy (DMD) after long-term treatment with eteplirsen. Clinical study 202 was an observational, open-label extension of the randomized, controlled study 201 assessing the safety and efficacy of eteplirsen in patients with DMD with a confirmed mutation in the gene amenable to correction by skipping of exon 51. Patients received once-weekly IV doses of eteplirsen 30 or 50 mg/kg. Upper extremity muscle biopsy samples were collected at combined study week 180, blinded, and assessed for dystrophin-related content by Western blot, Bioquant software measurement of dystrophin-associated

Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History Duchenne muscular dystrophy (DMD) is a rare, degenerative, X-linked genetic disease that results in progressive muscle loss and premature death, most commonly from respiratory or cardiac failure. DMD is primarily caused by whole exon deletions, resulting in a shift of the dystrophin mRNA reading frame that prevents production of functional dystrophin protein. Eteplirsen, a phosphorodiamidate morpholino oligomer (PMO), is designed to skip exon 51, restore the reading frame, and induce production of internally shortened dystrophin in patients with mutations amenable to such treatment. Describe lung function assessed throughout eteplirsen studies 201/202. Studies 201/202

Safety and efficacy of Eteplirsen in Patients with Duchenne Muscular Dystrophy: a systematic review and meta-analysis PROSPERO International prospective register of systematic reviews Print | PDF PROSPERO This information has been provided by the named contact for this review. CRD has accepted this information in good faith and registered the review in PROSPERO. The registrant confirms

Eteplirsen for the treatment of Duchenne muscular dystrophy: Quality of evidence concerns - An alternative viewpoint.

Eteplirsen Each month, subscribers to receive 5 to 6 well-documented monographs on drugs that are newly released or are in late phase 3 trials. The monographs are targeted to Pharmacy & Therapeutics Committees. Subscribers also receive monthly 1-page summary monographs on agents that are useful for agendas and pharmacy/nursing in-services. A comprehensive target drug utilization evaluation

Eteplirsen in the treatment of Duchenne muscular dystrophy Duchenne muscular dystrophy is a fatal neuromuscular disorder affecting around one in 3,500-5,000 male births that is characterized by progressive muscular deterioration. It is inherited in an X-linked recessive fashion and is caused by loss-of-function mutations in the gene coding for dystrophin, a cytoskeletal protein that stabilizes the plasma membrane of muscle fibers. In September 2016, the US Food and Drug Administration granted accelerated approval for eteplirsen (or Exondys 51), a drug that acts to promote dystrophin production by restoring the translational reading frame of through specific skipping of exon 51 in defective gene variants. Eteplirsen is applicable for approximately 14% of patients with mutations. This article

safety and efficacy of eteplirsen on Duchenne's muscular dystrophy: systematic review PROSPERO International prospective register of systematic reviews Print | PDF PROSPERO This information has been provided by the named contact for this review. CRD has accepted this information in good faith and registered the review in PROSPERO. The registrant confirms that the information supplied

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice This is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen, golodirsen, and casimersen in routine clinical practice . Participants in this study will have been prescribed eteplirsen, golodirsen, or casimersen commercially prior to entry into the study.

Eteplirsen Approved for Duchenne Muscular Dystrophy: The FDA Faces a Difficult Choice

Longitudinal effect of eteplirsen vs. historical control on ambulation in DMD. To continue evaluation of the long-term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer designed to skip DMD exon 51 in patients with Duchenne muscular dystrophy (DMD). Three-year progression of eteplirsen-treated patients was compared to matched historical controls (HC). Ambulatory DMD patients who were ≥7 years old and amenable to exon 51 skipping were randomized to eteplirsen (30/50mg/kg) or placebo for 24 weeks. Thereafter, all received eteplirsen on an open-label basis. The primary functional assessment in this study was the 6-Minute Walk Test (6MWT). Respiratory muscle function was assessed by pulmonary function testing (PFT). Longitudinal natural history data were used

Eteplirsen for the treatment of duchenne muscular dystrophy. 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). DMD boys aged 7 to 13 years, with confirmed deletions correctable by skipping exon 51 and ability to walk 200 to 400 m on 6 MWT, were randomized to weekly intravenous infusions of 30 or 50 mg/kg/wk eteplirsen or placebo for 24 weeks (n = 4/group). Placebo patients switched to 30 or 50 mg/kg eteplirsen (n=2/group) at week 25; treatment was open

, eteplirsen, golodirsen, delandistrogene moxeparvovec-rokl Drugs in clinical trials – Myotonic dystrophy 1 AD, anticipation congenital Family history, physical examination, EMG, muscle biopsy DMPK CTG repeats pathogenic & fully penetrant > 50; unstable premutation range 34-49 unstable Symptomatic & supportive therapy Definitive diagnosis by genetic testing Drugs in clinical trials – Myotonic dystrophy 2 AD

DMD Treatments includes prednisone, deflazacort, eteplirsen, golodirsen, delandistrogene moxeparvovec-rokl Drugs in clinical trials – Myotonic dystrophy 1 AD, anticipation congenital Family history, physical examination, EMG, muscle biopsy DMPK CTG repeats pathogenic & fully penetrant > 50; unstable premutation range 34-49 unstable Symptomatic & supportive therapy Definitive diagnosis by genetic