Brasil – quatorze camundongos foram tratados por 14 dias por via oral com óleo de peixe. Os animais tratados apresentaram menor necrose, menor inflamação, menor CK e redução de citocinas inflamatórias.

O resumo em inglês pode ser lido abaixo:

(Clinical Nutrition, 2012) Effects of fish oil containing eicosapentaenoic acid and docosahexaenoic acid on dystrophic mdx mice.

Adriana Fagagnolo Mauricio, Elaine Minatel, Humberto Santo Neto, Maria Julia Marques – Brazil

Abstract
Background and aims. In Duchenne muscular dystrophy (DMD) and in the mdx mouse model of DMD, the lack of dystrophin leads to muscle degeneration and inflammation contributes to progression of the disease. In this study, we evaluated the effects of commercially available fish oil containing EPA and docosahexaenoic acid (DHA) on mdx.

Methods. Mdx mice (14 days old) were treated with fish oil (FDC Vitamins; 0.002 g EPA and 0.001 g DHA) for 16 days by gavage. Control mdx mice received mineral oil (Nujol). Grip strength measurement was used for functional evaluation. The sternomastoid, diaphragm and biceps brachii muscles were removed and processed for histopathology and Western blot analysis.

Results. Fish oil decreased creatine kinase and myonecrosis. In all muscles studied, the inflammatory area was significantly reduced after treatment (18.0 ± 3.0% inflammatory area in untreated mdx mice vs 4.0 ± 1% in treated mdx mice). Fish oil protected against the loss of muscle strength. Fish oil significantly reduced the levels of TNF-α and the levels of 4-HNE-protein adducts (30 to 34% reduction for both) in all muscles studied.

Conclusions. Commercially available fish oil may be potentially useful to ameliorate dystrophic progression of skeletal muscles, deserving further clinical trials in DMD patients.

Fonte: http://distrofiamuscular.net/noticias.htm

Ucrânia – Vinte e sete pacientes com distrofia muscular de Duchenne, com idades variando de 4 a 27 anos receberam tratamento com um poll de células embrionárias retiradas de fetos sadios. A avaliação realizada 6 meses após o tratamento demosntrou uma melhora funcional e da qualidade de vida destes pacientes. Não há detalhes da qualtidade de células, vias de administração e a avaliação foi feita por um período muito curto, sem demonstrar que as células estavam presentes nos músculos.

O resumo em inglês pode ser lido abaixo:

(Annual Meeting American Neurological Association 2012) Fetal Stem Cells in Duchenne’s Muscular Dystrophy (DMD)

Nataliia S. Sych, Mariya O. Klunnyk, Olena V. Ivankova and Iryna G. Matiyaschuk; Kiev, Ukraine

Though modern methods can prolong life expectancy of DMD patients, but fetal stem cell therapy (FSCT) can be more promising. Goal of research was to study FSC efficacy in integrated treatment of DMD. Study included 27 male DMD patients aging 4-27. Diagnosis was confirmed clinically and on the basis of test results (high CPK and LDH), EMG, genetic testing, muscle biopsy. Functional capacity of patients was assessed on Total Functional Grade (TFG) scale, quality of life – on SF-36. Patients were examined before stem cell therapy treatment and 6 months after it. All patients underwent transplantation of hematopoietic and non-hematopoietic mesenchymal and ectodermal FSC harvested from germ layers of internal organs of 4-8 weeks old fetuses. FSCT resulted in improvements on TFG – from 8,5760,66 before the treatment to 7,7560,54 after the treatment, p<0,05. 0,5-point improvement was reported in 85,19% cases, 1-point – in 11,11%, and 1,5-point in 3,7%. FSCT also resulted in physical and psychoemotional improvement, higher self-esteem and spiritual realization. FSCT improved functional capacity and life quality of DMD patients, which proves that this method is very promising and should be researched and advanced.

Fonte: http://distrofiamuscular.net/noticias.htm

Brasil – agonistas AMPK/PPAR podem controlar o stress oxidativo. Nesta pesquisa os camundongos com distrofia muscular foram tratados com estes agonistas e submetidos a atividade física. Houve melhora do desempenho físico dos animais e redução do stress oxidativo.

Leiam mais sobre o artigo aqui.

Fonte: http://distrofiamuscular.net/noticias.htm

Itália – Os autores compararam os efeitos do tratamento com corticóides e com anti-inflamatórios não hormonais (aspirina, ibuprofeno e parecoxib) em camundongos com distrofia muscular. O resultado mostrou que estes anti-inflamatórios reduzem as alterações patológicas dos músculos, reduzem a inflamação e a necrose. O estudo da contratilidade não demonstrou efeito.

O resumo em inglês pode ser lido abaixo:

(Muscle Nerve, 46(5): 73-84, 2012) Inflammation in muscular dystrophy and the beneficial effects of non-steroidal anti-inflammatory drugs

Filippo Serra, Marco Quarta, Marta Canato Luana Toniolo Valeria De Arcangelis, Attilio Trotta, Lucia Spath, Lucia Monaco Carlo Reggiani Fabio Naro – Italy

Introduction: Glucocorticoids are the only drugs available for the treatment of Duchenne muscular dystrophy (DMD), but it is unclear whether their efficacy is dependent on their anti-inflammatory activity. Methods: To address this issue, mdx mice were treated daily with methylprednisolone and non-steroidal anti-inflammatory drugs (NSAIDs: aspirin, ibuprofen, parecoxib). Results: NSAID treatment was effective in ameliorating muscle morphology and reducing macrophage infiltration and necrosis. The percentage of regenerating myofibers was not modified by the treatments. The drugs were effective in reducing COX-2 expression and inflammatory cytokines, but they did not affect utrophin levels. The effects of the treatments on contractile performance were analyzed. Isometric tension did not differ in treated and untreated muscle, but the resistance to fatigue was decreased by treatment with methylprednisolone and aspirin. Conclusions: NSAIDs have a beneficial effect on mdx muscle morphology, pointing to a crucial role of inflammation in the progression of DMD.

Fonte: http://distrofiamuscular.net/noticias.htm

Canadá – sonolência diurna é uma das manifestações mais frequentes da distrofia miotônica. Vinte e quatro pacientes foram incluídos nestes estudo utilizando 20 mg de metilfenidato. Houve uma significativa melhora da sonolência diurna. A medicação tem efeitos colaterais conhecidos que também foram vistos neste estudo tais como palpitação, náuseas, vômitos e perda do apetite. Em alguns casos os efeitos colaterais como diarréa, nervosismo e irritabilidade levou a suspensão do medicamento. Apesar disto a droga foi considerada eficaz e uma boa alternativa para tratamento da sonolência diurna n distrofia miotônica do tipo 1.

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Fonte: http://distrofiamuscular.net/noticias.htm

Itália – os autores estudando 103 meninos com Duchenne identificaram 33 pacientes com Distúrbio do Déficit de Atenção e Hiperatividade. Destes 18 apresentavam também deficit intelectual, superior ao encontrado em Duchenne sem defict de atenção e hiperatividade. As mutações envolvendo os exons 45-55 e entre 62 e 63 são as que mais se relacionam ao deficit de atenção e hiperatividade.

O resumo em inglês pode ser lido abaixo:

(J Pediatr 2012;161:705-9) Attention Deficit Hyperactivity Disorder and Cognitive Function in Duchenne Muscular Dystrophy: Phenotype-Genotype Correlation

Marika Pane, Maria Elena Lombardo, Paolo Alfieri, Adele D’Amico, Flaviana Bianco, Gessica Vasco, Giorgia Piccini, Maria Mallardi, Domenico M. Romeo, Valeria Ricotti, Alessandra Ferlini, Francesca Gualandi, Stefano Vicari, Enrico Bertini, Angela Berardinelli, and Eugenio Mercuri – Italy

Objectives To assess attention deficit hyperactivity disorder (ADHD) in boys affected by Duchenne muscular dystrophy (DMD) and to explore the relationship with cognitive abilities and genetic findings. Study design Boys with DMD (n = 103; 4-17 years of age, mean: 12.6) were assessed using a cognitive test (Wechsler scales). Assessment of ADHD was based on the Diagnostic Statistical Manual, Fourth Edition, Text Revision criteria and on the long version of the Conners Parents and Teachers Rating Scales. Results ADHD was found in 33 of the 103 boys with DMD. Attention problems together with hyperactivity (17/33) or in isolation (15/33) were more frequent than hyperactivity alone, which was found in 1 patient. Intellectual disability (ID) was found in 27/103 (24.6%). Sixty-two of the 103 boys had no ID and no ADHD, 9 had ID but no ADHD, 14 had ADHD but no ID, and 18 had both. ADHD occurred more frequently in association with mutations predicted to affect Dp140 expression (exon 45-55) and in those with mutations predicted to affect all dystrophin product, including Dp71 (ie, those that have promoter region and specific first exon between exons 62 and 63 but were also relatively frequent). Conclusions Our results suggest that ADHD is a frequent feature in DMD. The risk of ADHD appears to be higher in patients carrying mutations predicted to affect dystrophin isoforms expressed in the brain and are known to be associated with higher risk of cognitive impairment.

Fonte: http://distrofiamuscular.net/noticias.htm

Brasil – os autores utilizaram a doxiciclina, um antibiótico da família da tetraciclina em camundongos com distrofia muscular; houve melhora das alterações patológicas do músculo cardíaco e músculos esqueléticos e aumento da força muscular nos animais tratados.

O resumo em inglês pode ser lido abaixo:

(Muscle Nerve 46: 400–406, 2012) DOXYCYCLINE AMELIORATES THE DYSTROPHIC PHENOTYPE OF SKELETAL AND CARDIAC MUSCLES IN mdx MICE

JULIANO ALVES PEREIRA, ANA PAULA TIEMI TANIGUTI, CINTIA MATSUMURA, MARIA JULIA MARQUES, HUMBERTO SANTO NETO – Brasil

ABSTRACT: Introduction: We examined whether doxycycline,an antibiotic member of the tetracycline family, improves the histopathology and muscle function in mdx mice, the experimental model of DMD. Methods: Doxycycline was administered for 36 days (starting on postnatal day 0) and for 9 months (starting at 8 months of age) in drinking water. Histopathological, biochemical (creatine kinase), and functional (forelimb muscle grip strength) parameters were evaluated in limb, diaphragm, and cardiac muscle. Results: Doxycycline significantly minimized the dystrophic phenotype of skeletal and cardiac muscles and improved forelimb muscle strength. The drug protected muscle fibers against myonecrosis and reduced inflammation. Furthermore, it slowed the progression of myocardial fibrosis. Conclusions: This study provides evidence that doxycycline may be a potential therapeutic agent for DMD.

Fonte: http://distrofiamuscular.net/noticias.htm

Autralia – neste congresso de especialistas em doenças neuromusculares que se realizará em Peth, Australia, serão apresentados mais de 300 pesquisas envolvendo diagnóstico, epidemiologia e tratamento clínico e experimental em doenças neuromusculares. Os resumos aqui incluídos são os principais relacionados com tratamento.

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Fonte: http://distrofiamuscular.net/noticias.htm

Suiça – a droga losartana, inibidora da angiotensina II já demostrou efeito em modelos experimentais de distrofia muscular. Nesta pesquisa foram utilizados camundongos com distrofia muscular congênita que foram tratados com L 158809, semelhante a losartana. Adroga diminui a formação de citocina inflamatória diminuindo as alterações patologicas dos músculos. Portanto neste modelo de distrofia experimental os inibidores da angiotensina II também são úteis.

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Fonte: http://distrofiamuscular.net/noticias.htm

USA – adolescentes com distrofia muscular de Duchenne em uso de corticóides apresentam retardo do crescimento. Neste estudo 39 adolescentes receberam por um ano o tratamento com hormônio do crescimento. Houve um significativo aumento do crescimento neste tratamento. Não houve alteração do peso, da força muscular e da função cardíaca e respiratória. Não ocorreram efeitos colaterais.

O resumo em inglês pode ser lido abaixo:

(Neuromuscular Disorders, 2012) Growth hormone treatment in boys with Duchenne muscular dystrophy and glucocorticoid-induced growth failure

Meilan M. Rutter, James Collins, Susan R. Rose, Jessica G. Woo, Heidi Sucharew, Hemant Sawnani, Kan N. Hor, Linda H. Cripe, Brenda L. Wong – USA

This study evaluated efficacy and safety of growth hormone treatment in Duchenne muscular dystrophy boys with glucocorticoid-induced growth failure. We reviewed 39 consecutive boys (average age 11.5 years; 32 ambulatory) treated with growth hormone for 1 year during a four-year period. Boys were on long-term daily deflazacort or prednisone (mean duration 5 ± 2.2 years; dosing regimen prednisone 0.75 mg/kg/day equivalent). Primary outcomes were growth velocity and height-for-age z-scores (height SD) at 1 year. Height velocity increased from 1.3 ± 0.2 to 5.2 ± 0.4 cm/year on growth hormone (p < 0.0001). Pre-growth hormone decline in height SD (−0.5 ± 0.2 SD/year) stabilized at height SD −2.9 ± 0.2 on growth hormone (p < 0.0001). The rate of weight gain was unchanged, at 2.8 ± 0.6 kg/year pre-growth hormone and 2.6 ± 0.7 kg/year at 1 year. Motor function decline was similar pre-growth hormone and at 1 year. Cardiopulmonary function was unchanged. Three experienced side effects. 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.

Fonte: http://distrofiamuscular.net/noticias.htm