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Duchenne Muscular Dystrophy

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1. Golodirsen (Vyondys 53) - Duchenne muscular dystrophy

Golodirsen (Vyondys 53) - Duchenne muscular dystrophy Drug Approval Package: Vyondys 53 (golodirsen) U.S. Department of Health and Human Services Search FDA Submit search Drug Approval Package: Vyondys 53 (golodirsen) Company: Sarepta Therapeutics, Inc. Application Number: 211970 Approval Date: 12/12/2019 Persons with disabilities having problems accessing the PDF files below may call (301) 796-3634 for assistance. FDA Approval Letter and Labeling (PDF) (PDF) FDA Application Review Files (PDF

2020 FDA - Drug Approval Package

2. Assisted standing for Duchenne muscular dystrophy. (Abstract)

Assisted standing for Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is the most common X-linked neuromuscular disorder. When boys with DMD reach the second decade of life, they lose their ability to walk and become wheelchair dependent. Standing devices and orthoses are considered to be an essential component in the therapy management of DMD. Clinical opinion and research from other neurological conditions highlight the proposed benefits of standing device use, however, its

2019 Cochrane

3. Ataluren (Translarna) - for the treatment of Duchenne muscular dystrophy (DMD)

Ataluren (Translarna) - for the treatment of Duchenne muscular dystrophy (DMD) Final Appraisal Recommendation Advice No: 0419 – March 2019 Ataluren (Translarna ® ) 125 mg, 250 mg and 1,000 mg granules for oral suspension Limited submission by PTC Therapeutics Ltd Additional note(s): • Please refer to the Summary of Product Characteristics for the full licensed indication. • AWMSG considered that ataluren (Translarna ® ) satisfied the AWMSG criteria for an ultra-orphan medicine. • AWMSG (...) recommend that ataluren (Translarna ® ) be used in accordance with Specialised Service Policy: CP118 Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene, and, National Institute of Health and Care Excellence highly specialised technology guidance HST3 for treating nonsense mutation DMD in children aged five years and older who can walk. In reaching the above recommendation AWMSG has taken account of the appraisal documentation prepared by the AWMSG

2019 All Wales Medicines Strategy Group

4. Interventions for preventing and treating cardiac complications in Duchenne and Becker muscular dystrophy and X-linked dilated cardiomyopathy. Full Text available with Trip Pro

Interventions for preventing and treating cardiac complications in Duchenne and Becker muscular dystrophy and X-linked dilated cardiomyopathy. The dystrophinopathies include Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), and X-linked dilated cardiomyopathy (XLDCM). In recent years, co-ordinated multidisciplinary management for these diseases has improved the quality of care, with early corticosteroid use prolonging independent ambulation, and the routine use of non-invasive (...) % confidence interval (CI) 0.07 to 15.77). This result is uncertain because of study limitations, indirectness and imprecision. In a non-randomised follow-up study, after 10 years, more participants who had received placebo from the beginning had reduced LVEF% (less than 45%). Adverse event rates were similar between the placebo and treatment groups (low-certainty evidence).A study comparing treatment with lisinopril versus losartan in 23 boys newly diagnosed with Duchenne cardiomyopathy showed that after

2018 Cochrane

5. Ataluren (Translarna) and Duchenne muscular dystrophy. No proof of efficacy and poorly documented harms

Ataluren (Translarna) and Duchenne muscular dystrophy. No proof of efficacy and poorly documented harms Prescrire IN ENGLISH - Spotlight ''Ataluren (Translarna°) and Duchenne muscular dystrophy. No proof of efficacy and poorly documented harms'', 1 January 2018 {1} {1} {1} | | > > > Ataluren (Translarna°) and Duchenne muscular dystrophy. No proof of efficacy and poorly documented harms Spotlight Every month, the subjects in Prescrire’s Spotlight. 100 most recent :  |   |    (...) |   |   |   |   |   |   |  Spotlight Ataluren (Translarna°) and Duchenne muscular dystrophy. No proof of efficacy and poorly documented harms FEATURED REVIEW Duchenne muscular dystrophy is a fatal, severely disabling disease that profoundly affects the lives of patients and their carers. But even in this very serious situation, there is no justification for proposing a drug with no proven efficacy beyond a placebo effect, and with poorly documented

2018 Prescrire

7. Interventions to prevent and treat corticosteroid-induced osteoporosis and prevent osteoporotic fractures in Duchenne muscular dystrophy. Full Text available with Trip Pro

Interventions to prevent and treat corticosteroid-induced osteoporosis and prevent osteoporotic fractures in Duchenne muscular dystrophy. Corticosteroid treatment is considered the 'gold standard' for Duchenne muscular dystrophy (DMD); however, it is also known to induce osteoporosis and thus increase the risk of vertebral fragility fractures. Good practice in the care of those with DMD requires prevention of these adverse effects. Treatments to increase bone mineral density include

2017 Cochrane

8. Respiratory Management of the Patient With Duchenne Muscular Dystrophy

Respiratory Management of the Patient With Duchenne Muscular Dystrophy Respiratory Management of the Patient With Duchenne Muscular Dystrophy | SUPPLEMENT ARTICLES | Pediatrics '); document.write(''); } function OAS_AD(pos) { if (OAS_version >= 11 && typeof(OAS_RICH)!='undefined') { OAS_RICH(pos); } else { OAS_NORMAL(pos); } } //--> Search for this keyword Source User menu Sections Sign up for highlighting editor-chosen studies with the greatest impact on clinical care. Respiratory Management (...) of the Patient With Duchenne Muscular Dystrophy Daniel W. Sheehan , David J. Birnkrant , Joshua O. Benditt , Michelle Eagle , Jonathan D. Finder , John Kissel , Richard M. Kravitz , Hemant Sawnani , Richard Shell , Michael D. Sussman , Lisa F. Wolfe Abstract In 2010, Care Considerations for Duchenne Muscular Dystrophy, sponsored by the Centers for Disease Control and Prevention, was published in Lancet Neurology , and in 2018, these guidelines were updated. Since the publication of the first set

2018 American Academy of Pediatrics

9. Respiratory Management of the Patient With Duchenne Muscular Dystrophy

Respiratory Management of the Patient With Duchenne Muscular Dystrophy PEDIATRICS Volume 142, number s2, October 2018:e20180333H SUPPLEMENT ARTICLE Respiratory Management of the Patient With Duchenne Muscular Dystrophy Daniel W. Sheehan, PhD, MD, a David J. Birnkrant, MD, b Joshua O. Benditt, MD, c Michelle Eagle, PhD, d Jonathan D. Finder, MD, e John Kissel, MD, f Richard M. Kravitz, MD, g Hemant Sawnani, MD, h Richard Shell, MD, i Michael D. Sussman, MD, j Lisa F. Wolfe, MD k a Department (...) as chairpersons for the Duchenne Muscular Dystrophy Care Considerations Respiratory Management Working Group, as convened by the Centers for Disease Control and Prevention, and drafted the initial manuscript; Drs Benditt, Eagle, Finder, Kissel, Kravitz, Sawnani, Shell, Sussman, and Wolfe all served on the Duchenne Muscular Dystrophy Care Considerations Respiratory Management Working Group, as convened by the Centers for Disease Control and Prevention, and contributed to the development of corresponding

2018 American Academy of Pediatrics

10. Deflazacort (Emflaza) - To treat patients age 5 years and older with Duchenne muscular dystrophy (DMD)

Deflazacort (Emflaza) - To treat patients age 5 years and older with Duchenne muscular dystrophy (DMD) Emflaza (deflazacort) Oral Tablets U.S. Department of Health and Human Services Search FDA Submit search Emflaza (deflazacort) Oral Tablets Emflaza Company: Gilead Sciences, Inc. Application No.: 208684 and 208685 Approval Date: 02/09/2017 Persons with disabilities having problems accessing the PDF files below may call (301) 796-3634 for assistance. (PDF) (PDF) (PDF) (PDF) (PDF) (PDF) (PDF

2017 FDA - Drug Approval Package

11. Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation

Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation Hind D, Parkin J, Whitworth V, Rex S, Young T, Hampson (...) , McMurchie H, Pallant L, Woods D, Freeman J, Lee E, Eagle M, Willis T, Muntoni F & Baxter P. Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation. Health Technology Assessment 2017; 21(27) Authors' objectives To assess the feasibility of recruiting boys with DMD to a randomised trial evaluating AT (primary objective) and to collect data from them; to assess how, and how well, the intervention and trial

2017 Health Technology Assessment (HTA) Database.

12. Emerging Drugs for Duchenne Muscular Dystrophy

Emerging Drugs for Duchenne Muscular Dystrophy Emerging Drugs for Duchenne Muscular Dystrophy | CADTH.ca CADTH Document Viewer Emerging Drugs for Duchenne Muscular Dystrophy Table of Contents Search this document Emerging Drugs for Duchenne Muscular Dystrophy June 2017 Summary Duchenne muscular dystrophy (DMD) is a rare and severe disorder that affects primarily young boys. It begins with progressive muscle weakness that evolves to loss of ambulation and further progresses to early morbidity (...) administrations. The oral route of administration may not be ideal in DMD patients who have trouble swallowing, although ataluren has the advantage of being available as granules for suspension. Eteplirsen needs to be infused intravenously once weekly, which could become a barrier, depending on accessibility and availability of treatment centres. References de los Angeles Beytía M, Vry J, Kirschner J. Drug treatment of Duchenne muscular dystrophy: Available evidence and perspectives. Acta Myol [Internet

2017 CADTH - Issues in Emerging Health Technologies

13. Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene

Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene Ataluren for treating Duchenne muscular Ataluren for treating Duchenne muscular dystroph dystrophy with a nonsense mutation in y with a nonsense mutation in the dystrophin gene the dystrophin gene Highly specialised technologies guidance Published: 20 July 2016 nice.org.uk/guidance/hst3 © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions (...) due regard to the need to eliminate unlawful discrimination, to advance equality of opportunity and to reduce health inequalities. Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible. Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (HST3) © NICE 2019. All rights reserved. Subject

2016 National Institute for Health and Clinical Excellence - Highly specialised technology

14. Emerging Drugs for Duchenne Muscular Dystrophy

Emerging Drugs for Duchenne Muscular Dystrophy Emerging Drugs for Duchenne Muscular Dystrophy | CADTH.ca CADTH Document Viewer Emerging Drugs for Duchenne Muscular Dystrophy Table of Contents Search this document Emerging Drugs for Duchenne Muscular Dystrophy June 2017 Summary Duchenne muscular dystrophy (DMD) is a rare and severe disorder that affects primarily young boys. It begins with progressive muscle weakness that evolves to loss of ambulation and further progresses to early morbidity (...) administrations. The oral route of administration may not be ideal in DMD patients who have trouble swallowing, although ataluren has the advantage of being available as granules for suspension. Eteplirsen needs to be infused intravenously once weekly, which could become a barrier, depending on accessibility and availability of treatment centres. References de los Angeles Beytía M, Vry J, Kirschner J. Drug treatment of Duchenne muscular dystrophy: Available evidence and perspectives. Acta Myol [Internet

2017 CADTH - Issues in Emerging Health Technologies

15. Ataluren (Translarna) - for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene

Ataluren (Translarna) - for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene All Wales Medicines Strategy Group (AWMSG) - ataluren (Translarna) | | Appraisal information Search and reports ataluren (Translarna®) Reference No. 940 Publication date: 19/08/2016 Appraisal information ataluren (Translarna®) 1000 mg granules for oral suspension ataluren (Translarna®) 125 mg granules for oral suspension ataluren (Translarna®) 250 mg granules (...) (Translarna®) for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older. At their meeting on 13th July 2016 AWMSG supported the adoption and implementation of this advice within NHS Wales (refer to the minutes of meetings). Welsh Government has subsequently agreed that the NICE HST advice for ataluren (Translarna®) should be implemented within NHS Wales. Refer to the NICE website for full guidance including any

2016 All Wales Medicines Strategy Group

16. Surgery for scoliosis in Duchenne muscular dystrophy. Full Text available with Trip Pro

Surgery for scoliosis in Duchenne muscular dystrophy. Scoliosis in patients with Duchenne muscular dystrophy (DMD) is usually progressive and is treated with surgery. However, it is unclear whether the existing evidence is sufficiently scientifically rigorous to support a recommendation for spinal surgery for most patients with DMD and scoliosis. This is an updated review, and an updated search was undertaken in which no new studies were found for inclusion.To determine the effectiveness

2015 Cochrane

17. Experiences of Women Who Have Had Carrier Testing for Duchenne Muscular Dystrophy and Becker Muscular Dystrophy During Adolescence Full Text available with Trip Pro

Experiences of Women Who Have Had Carrier Testing for Duchenne Muscular Dystrophy and Becker Muscular Dystrophy During Adolescence Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are X-linked recessive degenerative muscular conditions. Carrier testing is available to at-risk females. Though carrier testing is often offered to adolescent females, it raises ethical issues related to autonomy. This study aimed to address the impact of DMD/BMD carrier testing during

2018 Journal of Genetic Counseling

18. Health Care Transition Experiences of Males with Childhood-onset Duchenne and Becker Muscular Dystrophy: Findings from the Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet) Health Care Transitions and Other Life Experiences Survey Full Text available with Trip Pro

Health Care Transition Experiences of Males with Childhood-onset Duchenne and Becker Muscular Dystrophy: Findings from the Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet) Health Care Transitions and Other Life Experiences Survey Introduction: As the proportion of males with Duchenne muscular dystrophy (DMD) surviving into adulthood increases, more information is needed regarding their health care transition planning, an essential process for adolescents and young (...) adults with DMD. The objective of this study was to describe the health care transition experiences of a population of males living with Duchenne or Becker muscular dystrophy (DBMD). Methods: The eligible participants, identified through the Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet) surveillance project, were 16-31 years old and lived in Arizona, Colorado, Georgia, Iowa, or western New York (n=258). The MD STARnet Health Care Transitions and Other Life Experiences

2018 PLoS currents

19. Development and content validation of the Muscular Dystrophy Child Health Index of Life with Disabilities questionnaire for children with Duchenne muscular dystrophy. Full Text available with Trip Pro

Development and content validation of the Muscular Dystrophy Child Health Index of Life with Disabilities questionnaire for children with Duchenne muscular dystrophy. To develop a patient-reported outcome measure that comprehensively captures the health-related priorities of children with Duchenne muscular dystrophy (DMD).Children with DMD and their parents completed the iteratively revised versions of the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD), followed (...) , their parents, and health care professionals, and is ready for psychometric evaluation.The Muscular Dystrophy Child Health Index of Life with Disabilities (MDCHILD) is a new patient-reported outcome measure for Duchenne muscular dystrophy (DMD). The Priority Framework of Outcomes underpins the content for the MDCHILD. The MDCHILD incorporates the health-related priorities of males with DMD and their parents. The MDCHILD was deemed sensible by children, their parents, and health care professionals.© 2018 Mac

2018 Developmental Medicine and Child Neurology

20. Kyndrisa - Duchenne muscular dystrophy

Kyndrisa - Duchenne muscular dystrophy 30 Churchill Place ? Canary Wharf ? London E14 5EU ? United Kingdom An agency of the European Union Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5555 Send a question via our website www.ema.europa.eu/contact © European Medicines Agency, 2016. Reproduction is authorised provided the source is acknowledged. 24 June 2016 EMA/430531/2016 Rev 1 EMEA/H/C/003846 Questions and answers Withdrawal of the marketing authorisation application for Kyndrisa (...) (drisapersen) On 31 May 2016, BioMarin International Limited officially notified the Committee for Medicinal Products for Human Use (CHMP) that it wishes to withdraw its application for a marketing authorisation for Kyndrisa, for the treatment of Duchenne muscular dystrophy. What is Kyndrisa? Kyndrisa is a medicine that contains the active substance drisapersen. It was to be available as a solution for injection under the skin. What was Kyndrisa expected to be used for? Kyndrisa was expected to be used

2016 European Medicines Agency - EPARs

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