Targeting angiogenesis in Duchenne muscular dystrophy.

Targeting angiogenesis in Duchenne muscular dystrophy. Cell Mol Life Sci. 2019 Feb 15;: Authors: Podkalicka P, Mucha O, Dulak J, Loboda A Abstract Duchenne muscular dystrophy (DMD) represents one of the most devastating types of muscular dystrophies which affect boys already at early childhood. Despite the fact that the primary cause of the disease, namely the lack of functional dystrophin is known already for more than 30 years, DMD still remains an incurable disease. Thus, an enormous effort has been made during recent years to reveal novel mechanisms that could provide therapeutic targets for DMD, especially because glucocorticoids treatment acts mostly symptomatic and exerts many side effects, whereas the effectiveness of genetic approaches aiming at the restoration of functional dystrophin is under the constant debate. Taking into account that dystrophin expression is not restricted to muscle cells, but is present also in, e.g., endothelial cells, alterations in angiogenesis process have been proposed to have a significant impact on DMD progression. Indeed, already before the discovery of dystrophin, several abnormalities in blood vessels structure and function have been revealed, suggesting that targeting angiogenesis could be beneficial in DMD. In this review, we will summarize current knowledge about the angiogenesis status both in animal models of DMD as well as in DMD patients, focusing on different organs as well as age- and sex-dependent...
Source: Cellular and Molecular Life Sciences : CMLS - Category: Cytology Authors: Tags: Cell Mol Life Sci Source Type: research

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Our objective is to describe two genetically confirmed Oculopharyngeal Muscular Dystrophy (OPMD) patients with HMGCoARdase-antibody positive autoimmune necrotic myopathy (AINM) responsive to immunosuppressive treatment. Although clinicians have suspected muscular dystrophy patients are at increased risk of statin side-effects, to date no patients with genetically confirmed muscular dystrophy have been reported with HMGCoRdase-antibody positive AINM. Here we describe the clinical presentation, serology, muscle pathology and response to treatment in two patients with genetically confirmed OPMD who developed statin-associated AINM.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
In conclusion, our study demonstrated that elevated cumulative SBP or DBP was independently associated with increased risk of CVD in the Chinese population. Among participants with 15-year cumulative BP levels higher than the median, that is, 1970.8/1239.9 mmHg-year for cumulative SBP/DBP, which was equivalent to maintaining SBP/DBP level higher than 131/83 mmHg in 15 years, the CVD risk would increase significantly irrespective of whether or not the BP measurements at one examination was high. Our findings emphasize the importance of cumulative BP level in identifying individuals with high risk of CVD in the future. ...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
This update begins with muscle manifestations of coronavirus 2019. They may include myalgias and elevations in serum creatine kinase. It is unknown whether there is direct muscle invasion and how often the critically ill have muscle sequelae. Regarding autoimmune myopathies, a retrospective study of statin-induced necrotizing myopathy is covered. A relatively large proportion of patients had normal strength at presentation. Examples of dermatomyositis associated with immune checkpoint inhibitors are provided including one with cytokine storm. A report of juvenile dermatomyositis with severe abdominal complications is notew...
Source: Journal of Clinical Neuromuscular Disease - Category: Neurology Tags: Review Article Source Type: research
Duchenne muscular dystrophy (DMD), is the most common and severe neuromuscular disease affecting skeletal and cardiac muscle. Here, we showed that simvastatin both reversed pre ‐existing cardiac dysfunction and prevented its development during long‐term treatment of dystrophic mice. Simvastatin also prevented the progression of dystrophic cardiac fibrosis. Together, these findings indicate that simvastatin markedly improves cardiac health and function in dystrophic mic e, and therefore may provide a novel approach for treating cardiomyopathy in DMD patients. AbstractDuchenne muscular dystrophy (DMD), caused by absence ...
Source: Physiological Reports - Category: Physiology Authors: Tags: Original Research Source Type: research
Publication date: Available online 11 January 2019Source: Autoimmunity ReviewsAuthor(s): Céline Anquetil, Olivier Boyer, Nadège Wesner, Olivier Benveniste, Yves AllenbachAbstractOver the past few years, myositis-specific autoantibodies played an increasing role in the inflammatory idiopathic myositis definition. They became the critical immunological marker for immune-mediated necrotizing myopathy diagnosis (IMNM) since the paradigm switch from histological to serological criteria.This review is focused on the key role of the anti-signal recognition particle (anti-SRP) and the anti-3-Hydroxy-3-MethylGlutaryl-...
Source: Autoimmunity Reviews - Category: Allergy & Immunology Source Type: research
Publication date: Available online 11 January 2019Source: Autoimmunity ReviewsAuthor(s): Céline Anquetil, Olivier Boyer, Nadège Wesner, Olivier Benveniste, Yves AllenbachAbstractOver the past few years, myositis-specific autoantibodies played an increasing role in the inflammatory idiopathic myositis definition. They became the critical immunological marker for immune-mediated necrotizing myopathy diagnosis (IMNM) since the paradigm switch from histological to serological criteria.This review is focused on the key role of the anti-signal recognition particle (anti-SRP) and the anti-3-Hydroxy-3-MethylGlutaryl-...
Source: Autoimmunity Reviews - Category: Allergy & Immunology Source Type: research
In conclusion, in the Framingham Heart Study population, in the last 30 years, disease duration in persons with dementia has decreased. However, age-adjusted mortality risk has slightly decreased after 1977-1983. Consequences of such trends on dementia prevalence should be investigated. Recent Research on the Benefits of Exercise in Later Life https://www.fightaging.org/archives/2018/04/recent-research-on-the-benefits-of-exercise-in-later-life/ A sizable body of work points to the ability of older individuals to continue to obtain benefits through regular physical activity, and particularly in the case ...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
This study audited PCSK‐9 inhibitor therapy in 105 consecutive patients from two hospital centres–a university hospital (UH; n = 70) and a district general hospital (DGH; n = 35). Baseline characteristics including cardiovascular disease risk factors, NICE qualification criteria, efficacy and side effects were assessed. ResultsBaseline LDL‐C levels were similar in both centres. NICE criteria were met for 2.05 items in the whole study (UH patients 1.7 and DGH patients 2.7). District general hospital patients were more likely to have familial hypercholesterolaemia (89 vs 69%; P = .02);...
Source: International Journal of Clinical Practice - Category: Internal Medicine Authors: Tags: ORIGINAL PAPER Source Type: research
The US exhibits a remarkable pipeline of biopharmaceutical innovation, with 170 new drugs and biologics launched into the market between 2011 and 2015 and another 22 drugs approved in 2016. A striking feature of the pharmaceutical pipeline is the large percentage launched for the treatment of small “orphan” indications, defined by the Food and Drug Administration (FDA) as including fewer than, often many fewer than, 200,000 patients in the United States. Almost half (74) of the products approved by the FDA between 2011 and 2015 were for orphan indications, twice the number (36) approved during the same period b...
Source: Health Affairs Blog - Category: Health Management Authors: Tags: Costs and Spending Drugs and Medical Innovation Orphan Drug Act orphan drugs venture capitalism Source Type: blogs
From portable genome sequencers until genetic tests revealing distant relations with Thomas Jefferson, genomics represents a fascinatingly innovative area of healthcare. As the price of genome sequencing has been in free fall for years, the start-up scene is bursting from transformative power. Let’s look at some of the most amazing ventures in genomics! The amazing journey of genome sequencing Genome sequencing has been on an amazing scientific as well as economic journey for the last three decades. The Human Genome Project began in 1990 with the aim of mapping the whole structure of the human genome and sequencing ...
Source: The Medical Futurist - Category: Information Technology Authors: Tags: Genomics Personalized Medicine AI artificial intelligence bioinformatics cancer DNA dna testing DTC gc3 genetic disorders genetics genome sequencing personal genomics precision medicine Source Type: blogs
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