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![]() :: from the Institute A new tool to measure the daily life of patients At present, tools for evaluating the impact of treatments in neuromuscular disoders are mainly limited to strength measurement. Although this parameter is important, it is not sufficient to evaluate patients’ quality of life since it is not sufficiently comprehensive to evaluate a person’s activity as a whole: walking, sitting down, running, eating, going to bed, sleeping etc. This is why JY Hogrel and his team have devised a tool designed to measure the general activity of patients in their homes in order to reach parameters applicable to their daily life, and finally to be able to quantify any improvement during a particular therapy. In what context was the development of this tool considered necessary ? First we have realised that we had almost no instrument to measure the daily activity of patients in the case of systemic treatment. :: international Myostatin shows promise for Muscular Dystrophy Myostatin, a member of the TGF-b superfamily, is an endogenous inhibitor of muscle growth. Its structure and function are highly conserved across species including in humans. In the absence of myostatin, muscle growth is stimulated, at least partially, through the disinhibition of muscle progenitor cells. Myostatin has shown promising results as a potential therapeutic target for treating muscular dystrophy in animal studies, where its inhibition led to increased muscle mass and strength.
Hepatitis C drug may offer potential treatment strategy for Muscular Dystrophy In a study led by Dr. Jeff Molkentin at Cincinnati Children's Hospital Medical Centre, it was found that an investigational antiviral drug, Debio-025, currently undergoing human trials in Europe for treating Hepatitis C infections, may act as a potential treatment strategy to reduce muscle cell damage in Duchenne and other forms of muscular dystrophy (MD).
![]() Discover our selection of scientific and medical publications in the fields of myology and neuromuscular diseases: a summary of each publication aimed at the general reader, highlighting the main points of the article and the authors’ conclusions is provided.
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In this workshop on clinical trial development in the field of neuromuscular diseases, participants will learn how to develop a trial protocol from scratch. Lectures on study design, biostatistics, ICHGCP and European regulatory requirements will provide basic information. Ideally, participants will apply with a concrete idea of a planned study. During hands-on sessions there will be enough time to develop a protocol synopsis during the workshop. Members of the TREAT-NMD CTCC and other experts will assist participants in all aspects of the protocol development. > Programme
> Flyer and registration Form For further information or to express an interest please contact: annette.pohl(a)uniklinik-freiburg.de
![]() Recent publications from the Institute
Genethon: The first issue of Newsletter This month, Genethon published the first online issue of its Newletter in English.
Genethon’s Newsletter aims to keep you informed about its biotechnological activities, particularly in the field of research and the development of therapies for monogenic orphan diseases. Genethon’s Newsletter will be available every three months. Press release 12/02/2008 - A pharmacological therapeutic approach for certain sarcoglycanopathies A research team led by Dr Isabelle Richard (CNRS-FRE3087) from the Généthon laboratory financed by the AFM through Téléthon donations has just demonstrated the efficacy of a novel pharmacological strategy in the mouse for certain mutations of alpha-sarcoglycanopathy or LGMD2D, a recessive limb girdle dystrophy. This pharmacological strategy could be applied to other limb girdle muscular dystrophies characterised by the same type of genetic mutation. This work is published online in the review Human Molecular Genetics.
Funding calls MARIE CURIE Individual Fellowships
Intra-European Fellowships (IEF), International Incoming Fellowships (IIF) and International Outgoing Fellowships (IOF) The next call for Marie Curie Individual Fellowships is expected to come out on 19 March, with an expected deadline of 19 August. MARIE CURIE Initial Training Networks (ITN) The next call for Marie Curie Initial Training Networks (ITN) is expected to come out on 4 April with an expected deadline of 2 September. > For more information Books
Although the human genome has been sequenced, it can be difficult to find answers to seemingly simple questions about its characteristics. How many genes are there? Which genes are commonly associated with genetic diseases? How many DNA-binding proteins, mobile elements, or kinases are present? What are the major differences between human proteins and those of other species? This convenient handbook, written in question-and-answer format, allows researchers and teachers alike access to basic facts about the human genome.
> A Short Guide to the Human Genome
This is the first book of its kind that treats reproduction, cloning, stem cell research and regenerative medicine in an integrative manner. Touching on the science, social aspects, legal and ethical issues, and the current status of cloning, stem cell research and regenerative medicine, this self-contained book is an excellent source for introducing newcomers to the field or broadening the perspectives of experts and practitioners. It explains the diverse topics from a scientific angle, a social perspective, and as a natural business development. The coverage also includes the political and ethical issues as well as many other thought-provoking scenarios.
Recruitement
For further details contact the TREAT-NMD office (info(a)treat-nmd.eu), Michelle Eagle (michelle.eagle(a)btinternet.com) or Prof Kate Bushby (kate.bushby(a)ncl.ac.uk)
For further information please contact: Dr Simon Woods, 0191 243 0783, simon.woods(a)ncl.ac.uk.
Informal enquiries may be made to Dr Wood on tel. (01865) 272419 or on matthew.wood(a)dpag.ox.ac.uk. Further information can be found at: http://www.dpag.ox.ac.uk/job_opportunities
Molecular and cellular mechanisms underlying neuromuscular disorders
Please send your application, curriculum vitae and 2-3 referees addresses by email to : Dr. Jocelyn Laporte, E-mail : jocelyn(a)igbmc.u-strasbg.fr ![]() Discover our selection of scientific and medical publications in the field of myology and of neuromuscular diseases. The bimonthly Newsletter of the Institute of Myology keeps you up to date with developments in myology research, and presents a summary of the latest scientific, medical, political and associative news concerning neuromuscular diseases. You can access our Newsletter by connecting directly to the Institute of Myology website, or by subscribing. If you would prefer to receive this Newsletter in French, please click here.
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