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Muscular Dystrophy

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1. Muscular dystrophies

Muscular dystrophies Muscular dystrophies - Symptoms, diagnosis and treatment | BMJ Best Practice You'll need a subscription to access all of BMJ Best Practice Search  Muscular dystrophies Last reviewed: February 2019 Last updated: March 2019 Summary Duchenne muscular dystrophy (DMD) is highlighted as the most common and most rapidly progressive muscular dystrophy, with most patients losing the ability to walk by 12 years of age and requiring ventilatory support by 25 years of age. Before (...) for corrective surgery can be avoided, and the need for mechanical ventilation delayed, by early and aggressive use of corticosteroids. With a few exceptions, all generalised muscle disease can be managed using the principles for managing DMD. Definition Muscular dystrophies are progressive, generalised diseases of muscle, most often caused by defective or specifically absent glycoproteins (e.g., dystrophin) in the muscle membrane. All muscular dystrophies are characterised by ongoing degeneration and re

2019 BMJ Best Practice

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

2018 Cochrane

5. Characteristics of Japanese Patients with Becker Muscular Dystrophy and Intermediate Muscular Dystrophy in a Japanese National Registry of Muscular Dystrophy (Remudy): Heterogeneity and Clinical Variation (PubMed)

Characteristics of Japanese Patients with Becker Muscular Dystrophy and Intermediate Muscular Dystrophy in a Japanese National Registry of Muscular Dystrophy (Remudy): Heterogeneity and Clinical Variation Obtaining an adequate number of patients to conduct a natural history study for rare diseases such as Becker muscular dystrophy (BMD) is difficult.The present study used data from Remudy, a national registry for neuromuscular diseases in Japan, to conduct a phenotypic analysis of BMD.We (...) analyzed Remudy data of participants with dystrophinopathy. All participants who were aged 17 and older and were ambulant at age 13 were included in this study. Participants were divided into two groups: those with BMD who were ambulant at age 17, and those with intermediate muscular dystrophy (IMD) who lost ambulation by age 17. Frequent mutations were analyzed by age at ambulation, cardiopulmonary function, and genotype. For clinical comparisons, participants who were administered steroids were

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2018 Journal of neuromuscular diseases

6. 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. (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

8. Muscular dystrophies

Muscular dystrophies Muscular dystrophies - Symptoms, diagnosis and treatment | BMJ Best Practice You'll need a subscription to access all of BMJ Best Practice Search  Muscular dystrophies Last reviewed: February 2019 Last updated: March 2019 Summary Duchenne muscular dystrophy (DMD) is highlighted as the most common and most rapidly progressive muscular dystrophy, with most patients losing the ability to walk by 12 years of age and requiring ventilatory support by 25 years of age. Before (...) for corrective surgery can be avoided, and the need for mechanical ventilation delayed, by early and aggressive use of corticosteroids. With a few exceptions, all generalised muscle disease can be managed using the principles for managing DMD. Definition Muscular dystrophies are progressive, generalised diseases of muscle, most often caused by defective or specifically absent glycoproteins (e.g., dystrophin) in the muscle membrane. All muscular dystrophies are characterised by ongoing degeneration and re

2017 BMJ Best Practice

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. 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

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. 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

13. 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 (...) modulation) and givinostat (histone deacetylase inhibition) are in early stages of development. The evidence reviewed showed that, while these drugs increased the production of new dystrophin, they generally had limited impact on ambulation and physical function. Background DMD is an X-linked genetic disorder and is the most common form of muscular dystrophy in childhood, affecting one in 3,500 to 6,000 newborn males. 1,2 There are approximately 50,000 cases of DMD worldwide. 3 In 2015, it was estimated

2017 CADTH - Issues in Emerging Health Technologies

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 (...) modulation) and givinostat (histone deacetylase inhibition) are in early stages of development. The evidence reviewed showed that, while these drugs increased the production of new dystrophin, they generally had limited impact on ambulation and physical function. Background DMD is an X-linked genetic disorder and is the most common form of muscular dystrophy in childhood, affecting one in 3,500 to 6,000 newborn males. 1,2 There are approximately 50,000 cases of DMD worldwide. 3 In 2015, it was estimated

2017 CADTH - Issues in Emerging Health Technologies

15. 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

16. 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

17. Development and content validation of the Muscular Dystrophy Child Health Index of Life with Disabilities questionnaire for children with Duchenne muscular dystrophy. (PubMed)

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 (...) by a cognitive interview to develop a pilot version of a new measure. Multidisciplinary health care professionals completed an item-by-item analysis of the measure and a 14-item sensibility questionnaire. Minimum content validity ratio for each item of the new measure and the mean score (0-7) for the items of the sensibility questionnaire were calculated.The CPCHILD underwent changes over 19 interviews with children and their parents, resulting in the pilot Muscular Dystrophy Child Health Index of Life

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2018 Developmental Medicine and Child Neurology

18. 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.

19. 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.

20. Serum Creatinine Distinguishes Duchenne Muscular Dystrophy from Becker Muscular Dystrophy in Patients Aged ≤3 Years: A Retrospective Study (PubMed)

Serum Creatinine Distinguishes Duchenne Muscular Dystrophy from Becker Muscular Dystrophy in Patients Aged ≤3 Years: A Retrospective Study Here, we investigated correlations between serum creatinine (SCRN) levels and clinical phenotypes of dystrophinopathy in young patients. Sixty-eight patients with dystrophinopathy at the Neuromuscular Clinic, The First Affiliated Hospital, Sun Yat-sen University, were selected for this study. The diagnosis of dystrophinopathy was based on clinical (...) mild Becker muscular dystrophy (BMD) (p < 0.001) and the lowest in patients with Duchenne muscular dystrophy (DMD) (p < 0.01) and were significantly higher in patients carrying in-frame mutations than in patients carrying out-of-frame mutations (p < 0.001). SCRN level cutoff values for identifying mild BMD [18 µmol/L; area under the curve (AUC): 0.947; p < 0.001] and DMD (17 µmol/L; AUC: 0.837; p < 0.001) were established. These results suggest that SCRN might be a valuable biomarker

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2017 Frontiers in neurology

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