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Duchenne Muscular Dystrophy

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1. Assisted standing for Duchenne muscular dystrophy. (PubMed)

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

2. Interventions for preventing and treating cardiac complications in Duchenne and Becker muscular dystrophy and X-linked dilated cardiomyopathy. (PubMed)

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

4. 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 Journals Library An error occurred retrieving content to display, please try again. >> >> >> Page Not Found Page not found (404) Sorry - the page you requested could not be found. Please choose a page (...) from the navigation or try a website search above to find the information you need. >> >> >> >> Issue {{metadata .Issue }} Toolkit 1)"> 0)"> 1)"> {{metadata.Title}} {{metadata.Headline}} This feasibility study showed whilst aquatic therapy for Duchenne muscular dystrophy is valued, its delivery in tertiary centres poses problems and further intervention development is needed before a full RCT. {{author}} {{($index , , , , , , , , , , , , , , , , , , , , & . Daniel Hind 1, * , James Parkin 1

2017 NIHR HTA programme

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

6. Interventions to prevent and treat corticosteroid-induced osteoporosis and prevent osteoporotic fractures in Duchenne muscular dystrophy. (PubMed)

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

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2017 Cochrane

7. 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

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. 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.

10. Corticosteroids for the treatment of Duchenne muscular dystrophy. (PubMed)

Corticosteroids for the treatment of Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy of childhood. Untreated, this incurable disease, which has an X-linked recessive inheritance, is characterised by muscle wasting and loss of walking ability, leading to complete wheelchair dependence by 13 years of age. Prolongation of walking is a major aim of treatment. Evidence from randomised controlled trials (RCTs) indicates that corticosteroids

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2016 Cochrane

11. 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

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. Idebenone (Raxone) for Duchenne muscular dystrophy - first line

Idebenone (Raxone) for Duchenne muscular dystrophy - first line Idebenone (Raxone) for Duchenne muscular dystrophy – first line Idebenone (Raxone) for Duchenne muscular dystrophy – first line NIHR HSRIC Record Status This is a bibliographic record of a published health technology assessment. No evaluation of the quality of this assessment has been made for the HTA database. Citation NIHR HSRIC. Idebenone (Raxone) for Duchenne muscular dystrophy – first line. Birmingham: NIHR Horizon Scanning (...) Research&Intelligence Centre. Horizon Scanning Review. 2016 Authors' objectives Duchenne muscular dystrophy is an inherited condition that mainly affects boys and causes muscle weakness. Most people with Duchenne muscular dystrophy are diagnosed by the age of 5 years, and need to use a wheelchair by the age of 12 years. Many will face severe health problems by their late teens as their heart muscle and chest muscles become weaker, eventually affecting their breathing. Idebenone is a new drug

2016 Health Technology Assessment (HTA) Database.

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

Eteplirsen for Duchenne muscular dystrophy in patients amenable to exon 51 skipping Eteplirsen for Duchenne muscular dystrophy in patients amenable to exon 51 skipping Eteplirsen for Duchenne muscular dystrophy in patients amenable to exon 51 skipping NIHR HSRIC Record Status This is a bibliographic record of a published health technology assessment. No evaluation of the quality of this assessment has been made for the HTA database. Citation NIHR HSRIC. Eteplirsen for Duchenne muscular (...) dystrophy in patients amenable to exon 51 skipping. Birmingham: NIHR Horizon Scanning Research&Intelligence Centre. Horizon Scanning Review. 2016 Authors' conclusions Duchenne muscular dystrophy is an inherited condition that causes muscle weakness. It affects mainly boys and is caused by a mutation (a change) in the gene that makes dystrophin. Dystrophin is important for protecting muscles from stress and damage during activity. Most people with Duchenne muscular dystrophy are diagnosed by the age of 5

2016 Health Technology Assessment (HTA) Database.

15. Variability and trends in corticosteroid use by male United States participants with Duchenne muscular dystrophy in the Duchenne Registry. (PubMed)

Variability and trends in corticosteroid use by male United States participants with Duchenne muscular dystrophy in the Duchenne Registry. Treatment options for Duchenne muscular dystrophy remain limited, although consensus treatment guidelines recommend corticosteroid use.This retrospective analysis assessed corticosteroid use in ambulatory and nonambulatory US males with Duchenne, age 35 and under, or Becker muscular dystrophy, who enrolled in The Duchenne Registry from 2007 to 2016 (formerly (...) for never initiating therapy were that corticosteroids were not prescribed or recommended and concerns about side effects. Corticosteroid use was maximal at age 8 (84% on corticosteroids) and gradually declined from age 10 to 19. The primary reasons for corticosteroid discontinuation were problems with side effects (65%) or not enough benefit (28%). Average doses of corticosteroids were below recommended doses. In the 159 responses with Becker muscular dystrophy, 20% were currently using

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2019 BMC Neurology

16. Corticosteroid treatment of duchenne muscular dystrophy

Corticosteroid treatment of duchenne muscular dystrophy Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy | Neurology Advertisement Search for this keyword Main menu User menu Search Search for this keyword The most widely read and highly cited peer-reviewed neurology journal Share February 02, 2016 ; 86 (5) Special Article Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy Report of the Guideline Development (...) .), Loma Linda University Medical Center, CA; and Departments of Pediatric and Neurology/Neurosurgery (M.O.), McGill University, Montréal, Canada. Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy David Gloss , Richard T. Moxley , Stephen Ashwal , Maryam Oskoui Neurology Feb 2016, 86 (5) 465-472; DOI: 10.1212/WNL.0000000000002337 Citation Manager Formats Make Comment See Comments Downloads 5504 Share Abstract Objective: To update the 2005 American Academy

2016 American Academy of Neurology

17. 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

18. Maximizing the Benefit of Life-Saving Treatments for Pompe Disease, Spinal Muscular Atrophy, and Duchenne Muscular Dystrophy Through Newborn Screening: Essential Steps. (PubMed)

Maximizing the Benefit of Life-Saving Treatments for Pompe Disease, Spinal Muscular Atrophy, and Duchenne Muscular Dystrophy Through Newborn Screening: Essential Steps. Newborn screening (NBS) identifies infants with specific congenital disorders for which earlier intervention cannot only prevent a lifetime of chronic disability but also, most importantly, save lives. In this article, we discuss complexities associated with NBS processes in the United States, with a focus on challenges (...) in neuromuscular disorders.As new interventions for neuromuscular disorders become available, the clinical community must prepare to overcome the challenges of adding new diseases to screening panels and understand the rigorous evidence review at the federal level and the complex process of state-level implementation. In this regard, NBS programs for Pompe disease and spinal muscular atrophy can guide the path of Duchenne muscular dystrophy and other neuromuscular disorders as future candidates for NBS.The

2019 JAMA neurology

19. Experiences of Women Who Have Had Carrier Testing for Duchenne Muscular Dystrophy and Becker Muscular Dystrophy During Adolescence (PubMed)

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

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2018 Journal of Genetic Counseling

20. 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 (PubMed)

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

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2018 PLoS currents

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