|1.||Duchenne Muscular Dystrophy (Muscular Dystrophy, Becker)
|2.||Muscular Dystrophies (Muscular Dystrophy)
|4.||Limb-Girdle Muscular Dystrophies (Limb-Girdle Muscular Dystrophy)
|5.||Cystic Fibrosis (Mucoviscidosis)
|1.||Matsuo, Masafumi: 41 articles (01/2016 - 01/2002)|
|2.||Takeshima, Yasuhiro: 35 articles (01/2016 - 01/2002)|
|3.||Aartsma-Rus, Annemieke: 34 articles (06/2015 - 10/2002)|
|4.||Muntoni, Francesco: 33 articles (05/2015 - 07/2002)|
|5.||Takeda, Shin'ichi: 33 articles (05/2015 - 03/2002)|
|6.||Chamberlain, Jeffrey S: 32 articles (12/2014 - 03/2002)|
|7.||Duan, Dongsheng: 30 articles (12/2015 - 03/2002)|
|8.||Davies, Kay E: 30 articles (12/2015 - 04/2002)|
|9.||Hoffman, Eric P: 30 articles (09/2015 - 08/2002)|
|10.||Flanigan, Kevin M: 30 articles (06/2015 - 04/2003)|
02/01/2014 - "When transplanted into mdx mice models for Duchenne muscular dystrophy (DMD), the proposed muscle patches led to the formation of a significantly greater number of dystrophin-positive muscle fibers, indicating that dystrophin replacement and myogenesis is achievable in vivo with this approach. "
07/01/1997 - "Hopes ran high that a cure for Duchenne muscular dystrophy (DMD) would quickly follow the discovery of dystrophin by Lou Kunkel and his group in the 1980's. "
01/01/2007 - "Duchenne muscular dystrophy (DMD) is an X-linked recessive disease caused by mutations of dystrophin gene, there is no effective treatment for this disorder at present. "
03/01/2004 - "Despite a wealth of recent information about the molecular basis of Duchenne muscular dystrophy (DMD), there is no effective treatment for this condition because the mechanism how dystrophin deficiency produces the muscle fiber degradation is unknown. "
07/01/2012 - "Antisense oligonucleotide (AON)-mediated exon skipping to restore dystrophin expression in Duchenne muscular dystrophy (DMD) therapy shown promise in a number of human clinical trials. "
|2.||Creatine Kinase (Creatine Phosphokinase)IBA
06/01/1989 - "While diagnosis is still unacceptably late in most cases, it can be improved if all boys with this pattern of developmental delay are screened for Duchenne muscular dystrophy by measurement of creatine kinase activity."
02/01/1982 - "The mean serum creatine kinase levels before and after exercise, as well as the mean increases, were found to be significantly greater in Duchenne muscular dystrophy carriers than in normal control subjects."
01/01/1987 - "Data obtained from simultaneous determinations of serum creatine-kinase levels and estimation of ultrasound attenuation values in muscles greatly improved the detection of obligate carriers of Duchenne muscular dystrophy than when only one of these methods was employed alone. "
01/01/1986 - "Screening for Duchenne muscular dystrophy: an improved screening test for creatine kinase and its application in an infant screening program."
01/01/1983 - "Improved detection of Duchenne muscular dystrophy heterozygotes using discriminant analysis of creatine kinase levels."
|3.||Prednisolone (Predate)FDA LinkGeneric
01/01/2014 - "Of the numerous clinical trials for Duchenne muscular dystrophy, only the corticosteroid prednisolone has shown potential for temporal improvement in motor ability. "
12/01/2013 - "We evaluated the long-term efficacy of prednisolone (PSL) therapy for prolonging ambulation in Japanese patients with genetically confirmed Duchenne muscular dystrophy (DMD). "
01/01/2008 - "Remission of clinical signs in early Duchenne muscular dystrophy on intermittent low-dosage prednisolone therapy. "
01/01/2002 - "Remission of clinical signs in early duchenne muscular dystrophy on intermittent low-dosage prednisolone therapy."
01/01/2014 - "In this study, the effects of prednisolone on intellectual ability are examined in 29 cases of Duchenne muscular dystrophy because little information has been reported. "
|4.||Prednisone (Sone)FDA LinkGeneric
08/01/2002 - "The recent introduction of pharmaceutical agents such as prednisone has shown great promise in delaying the progression of Duchenne muscular dystrophy but there remains a need to develop more long-term therapeutic interventions. "
01/01/2010 - "One study assessed the efficacy of prednisone treatment in 103 boys with Duchenne muscular dystrophy. "
11/01/2012 - "Although prednisone has never been formally approved for use in Duchenne muscular dystrophy (DMD) by regulatory agencies, its efficacy has been confirmed in trials dating from the 1980s. "
08/02/2011 - "To perform a double-blind, randomized study comparing efficacy and safety of daily and weekend prednisone in boys with Duchenne muscular dystrophy (DMD). "
08/02/2011 - "Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy."
|5.||Adrenal Cortex Hormones (Corticosteroids)IBA
05/01/2009 - "Corticosteroids are effective in improving motor function in Duchenne muscular dystrophy (DMD) patients within 6 months-2 years of treatment initiation, but there is as yet no consensus on which treatment scheme is the best. "
05/01/2008 - "Are corticosteroids effective in Duchenne muscular dystrophy."
02/01/2012 - "There is no current cure for Duchenne muscular dystrophy (DMD), and palliative and prophylactic interventions to improve the quality of life of patients remain limited, with the exception of corticosteroids. "
05/01/2003 - "This study of 33 boys with Duchenne muscular dystrophy determined retrospectively the incidence of vertebral fractures particularly after initiation of corticosteroids. "
01/01/1995 - "Several studies have demonstrated the slowing effect of corticosteroids on the decline of muscle strength in Duchenne muscular dystrophy (DMD). "
|6.||Growth Hormone (Somatotropin)IBA
12/01/2012 - "In this first comprehensive report of growth hormone in Duchenne muscular dystrophy, growth hormone improved growth at 1 year, without detrimental effects observed on neuromuscular and cardiopulmonary function."
12/01/2012 - "This study evaluated efficacy and safety of growth hormone treatment in Duchenne muscular dystrophy boys with glucocorticoid-induced growth failure. "
01/01/2013 - "To describe a biochemical growth hormone (GH) deficiency and to evaluate therapeutic result in a six-year-old male with Becker muscular dystrophy (BMD). "
01/01/2013 - "Growth hormone deficiency in a patient with becker muscular dystrophy: a pediatric case report."
12/01/2012 - "Growth hormone treatment in boys with Duchenne muscular dystrophy and glucocorticoid-induced growth failure."
02/15/2004 - "Utrophin has been studied extensively in recent years in an effort to find a cure for Duchenne muscular dystrophy. "
01/01/2004 - "These results have important implications for future studies aiming to effect therapeutic utrophin up-regulation in Duchenne muscular dystrophy patients."
10/01/2002 - "Previous studies on transgenic mice indicate that upregulation of utrophin protein may offer a potential treatment strategy for Duchenne muscular dystrophy. "
01/01/2016 - "Upregulation of utrophin A is an attractive therapeutic strategy for treating Duchenne muscular dystrophy (DMD). "
06/01/2015 - "SMT C1100 is a small molecule utrophin modulator in development to treat Duchenne muscular dystrophy. "
|8.||Proteins (Proteins, Gene)IBA
01/01/2015 - "Transgenic overexpression of SSPN in Duchenne muscular dystrophy mice (mdx(TG)) improved cardiomyofiber cell adhesion, sarcolemma integrity, cardiac functional parameters, as well as increased expression of compensatory transmembrane proteins that mediate attachment to the extracellular matrix. "
03/01/1990 - "In the present study, degradation rates of individual intracellular proteins of normal human skin fibroblasts and skin fibroblasts from patients with Duchenne muscular dystrophy were compared. "
04/01/1989 - "Studies of water permeability and proteins of erythrocyte membranes in patients with Duchenne muscular dystrophy."
12/01/1988 - "More recent advances have been in the cloning and study of the involved proteins in disorders such as Duchenne muscular dystrophy. "
01/01/2014 - "As opposed to classical dystrophies, such as Duchenne muscular dystrophy, the dysregulation of various matricellular proteins appears to be a distinct feature of the early progression of DyW pathology. "
01/01/2003 - "A preclinical screening for prompt-to-use drugs that are safer than steroids and beneficial in Duchenne muscular dystrophy was performed. "
10/01/2002 - "Steroids in Duchenne muscular dystrophy: from clinical trials to genomic research."
04/01/1993 - "The study was performed as a preliminary to an investigation on the effect of anabolic steroids on skeletal muscle regeneration in the mdx mouse, an animal model used for the study of Duchenne muscular dystrophy. "
06/01/2015 - "With the exception of steroids for Duchenne muscular dystrophy, bracing remains the only reliable nonoperative intervention available. "
01/01/2015 - "Compared to males without a Duchenne muscular dystrophy family history, males with known family history were half as likely to use steroids. "
|10.||RNA Precursors (Precursor, mRNA)IBA
08/01/2007 - "Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo."
05/01/1996 - "Modulation of pre-mRNA splicing in the Duchenne muscular dystrophy gene."
02/01/2014 - "In particular, the use of AONs for exon skipping, by targeting pre-mRNA, is proving to be a highly promising therapy for some genetic disorders like Duchenne muscular dystrophy and spinal muscular atrophy. "
01/01/2016 - "Antisense oligonucleotide (AON)-mediated splice modulation appears to be a powerful approach to correct the consequences of such mutations at the pre-mRNA level , as demonstrated by promising results in clinical trials for several inherited disorders like Duchenne muscular dystrophy, hypercholesterolemia and various types of cancer. "
01/01/2011 - "Pre-mRNA targeting is currently used both as a research tool, e.g., in models for motor neuron disease, and in clinical trials for Duchenne muscular dystrophy and amyotrophic lateral sclerosis. "
|1.||Artificial Respiration (Mechanical Ventilation)
01/01/2012 - "Mechanical ventilation and cardioprotective therapy have significantly improved the prognosis and quality of life of patients with Duchenne muscular dystrophy (DMD). "
10/01/2011 - "Mechanical ventilation (MV) and cardiac protective therapy have improved the prognosis and quality of life of patients with Duchenne muscular dystrophy (DMD). "
08/01/2010 - "Mechanical ventilation has improved survival in patients with Duchenne muscular dystrophy (DMD). "
04/01/2009 - "Longitudinal determination of TT MUS in patients with Duchenne muscular dystrophy may help to justify the extent of need for mechanical ventilation."
04/01/2010 - "This paper is a report of a study from a patient perspective of the life experiences with home mechanical ventilation among young men with Duchenne muscular dystrophy. "
|2.||Transplantation (Transplant Recipients)
06/01/2010 - "The CGD was cured after the second transplantation, but 2.5 years later he was diagnosed with Duchenne muscular dystrophy (DMD). "
10/01/2009 - "[Experimental study of treating Duchenne muscular dystrophy with myoblast transplantation]."
12/01/2004 - "The aim of this study was to investigate the clinical feasibility of adult stem cell transplantation for lethal mono-gene inherited disease, Duchenne muscular dystrophy (DMD). "
01/01/2001 - "The clinical trials of myoblast transplantation in Duchenne Muscular Dystrophy (DMD) patients produced disappointing results. "
03/01/1993 - "Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy."
|3.||Tissue Therapy (Cell Therapy)
01/01/2015 - "Cell therapy in Duchenne muscular dystrophy treatment: clinical trials overview."
09/01/2013 - "In the present review, we describe the different types of myogenic stem cells and their possible use for the progression of cell therapy in Duchenne muscular dystrophy. "
02/01/2010 - "DYS-HAC-iPS cells: the combination of gene and cell therapy to treat duchenne muscular dystrophy."
12/01/2008 - "Another new "super muscle stem cell" leaves unaddressed the real problems of cell therapy for duchenne muscular dystrophy."
12/01/2006 - "CSCI/RCPSC Henry Friesen Lecture: Cell therapy for Duchenne muscular dystrophy."
01/01/2015 - "Antisense oligonucleotide (AO)-mediated exon-skipping therapeutics shows great promise for Duchenne muscular dystrophy (DMD) patients. "
06/01/2015 - "Duchenne Muscular Dystrophy: Advances in Molecular Genetics and Changing Strategies in Diagnosis, Prevention and Therapeutics."
07/01/2011 - "The identification of the Duchenne muscular dystrophy gene and protein in the late 1980s led to high hopes of rapid translation to molecular therapeutics. "
08/01/2010 - "PRO-051 (GSK-2402968), being developed by GlaxoSmithKline plc, under license from Leiden University Medical Center and Prosensa Therapeutics BV, is a 2'-O-methyl phosphorothioate antisense oligonucleotide for the potential treatment of Duchenne muscular dystrophy (DMD). "
05/01/2009 - "The availability of animal models for Duchenne muscular dystrophy has led to extensive preclinical research on potential therapeutics. "
|5.||Intermittent Positive-Pressure Ventilation
09/01/2006 - "The present study aimed to assess the impact of diurnal mouthpiece intermittent positive pressure ventilation (MIPPV) as the extension of the nasal intermittent positive pressure ventilation (NIPPV) in Duchenne muscular dystrophy (DMD). "
08/01/2010 - "In this retrospective study, patients with Duchenne muscular dystrophy who received glucocorticoid therapy were compared with those who did not for ages at wheelchair dependence and when beginning part-time (nocturnal) and continuous noninvasive intermittent positive pressure ventilation (NIV). "
09/01/2005 - "We have been introducing the intermittent positive pressure ventilation (IPPV) therapy in Duchenne muscular dystrophy (DMD) since 1990. "
09/01/2005 - "[Effect of intermittent positive pressure ventilation on life-span and causes of death in Duchenne muscular dystrophy]."
06/01/2002 - "To quantitate prolongation of survival for patients with Duchenne muscular dystrophy with the use of noninvasive intermittent positive-pressure ventilation (IPPV) with and without access to a protocol involving mechanically assisted coughing. "