Newsletter :: Institut de Myologie
#13
Bimonthly Newsletter - February/March 2008  
:: Research highlights
  :: from the Institute
  A new tool to measure daily life of patients JY Hogrel
  :: international
  Myostatin shows promise for Muscular Dystrophy
  Hepatitis C drug may offer potential treatment strategy for Muscular Dystrophy
:: Latest research highlights
:: Agenda
:: In brief
  Publications
  Généthon : First Newsletter issue
  Press release
  Funding calls
  Books
  Recruitement
:: Subscription
Edito
Myology 2008 is approaching quickly! With more than 1000 inscriptions and over 500 abstracts submitted, the congress promises to be filled with many fruitful discussions and presentations. Among the developments for this Myology 2008 edition is the advocacy to approach both themes of the conference each day -fundamental and therapeutic- across transverse subjects or pathology. Professor André Langaney (National Museum of Natural History in Paris and University of Geneva) will give the opening lecture, which will be followed by the welcome cocktail. On the second day, stem cells will be the topic of discussion with talks from renowned myologists such as Shin’ichi Takeda and Giulio Cossu, to name but a few. The sessions of day 3 will include presentations covering the heart, laminopathies and dystrophinopathies. Kenneth Chien and Denis Duboc will commence the day with two plenary lectures on stem
Myology 2008 header
cells in the heart and clinical trials in cardio-myopathies linked to neuromuscular diseases, respectively. Motor neurone diseases and structural myopathies will be the highlights of day 4. The 5th and last day of Myology 2008 will be consecrated to gene-based therapeutics and the evaluation of the neuromuscular patient. This final day will also coincide with the 1st day of the 5th international congress of rehabilitation in neuromuscular diseases, which will be opened by Laurence Tiennot-Herment, and Wahiba Boucharef.
With such an exciting scientific programme gathering the top notches in myology, not to mention the exceptional location of the Parc Chanot Convention Centre in Marseille, we’re sure that Myology 2008 will be enjoyed by all!
 
RNC
   Research highlights

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:: from the Institute


JY Hogrel
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.
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:: international


John Hopkins school of Medecine logo
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.

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cincinnati children's hospital logo
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).

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   Latest research highlights

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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.

  • Muscular dystrophy research and treatment challenged - Read
  • A new type 1 myotonic dystrophy mouse model - Read
  • Preventing fibrosis in mdx mice with a novel anti-fibrotic drug-halofuginone - Read
  • Targeting astrocytes slows disease progression in amyotrophic lateral sclerosis - Read
  • Two new mutations in the fukutin gene causing Walker-Warburg syndrome - Read
  • Potential use of negamycin to induce readthrough of stop codon mutations - Read
  • Vascular endothelial growth factor improves myoblast survival - Read
  • Unexpected protein interaction suggests new ALS drug target - Read
  • Type 1 myotonic muscular dystrophy is associated with induced NKX2-5 expression - Read
  • Treating muscular dystrophy with stem cells - Read
  • Pilot study of ACE inhibitor in McArdle's disease - Read
  • First Successful Clinical Study with RNA-based Therapeutic PRO051 in Duchenne Muscular Dystrophy - Read

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   Agenda

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  • 14th International Conference on Prenatal Diagnosis and Therapy
    1-4 June 2008, Vancouver, Canada

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  • Clinical Trials in Neuromuscular Diseases
    19-21 June 2008, Freiburg, Germany
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


  • 20th International Congress of Genetics
    12-17 July 2008, Berlin, Germany

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  • 12th congress of the European Federation of Neurological Societies - EFNS 2008
    23-26 August 2008, Madrid, Spain
>>> Access the complete list of upcoming conferences and meeting.


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   In Brief

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Recent publications from the Institute

  • Allamand V, Bidou L, Arakawa M, Floquet C, Shiozuka M, Paturneau-Jouas M, Gartioux C, Butler-Browne GS, Mouly V, Rousset JP, Matsuda R, Ikeda D, Guicheney P.
    Drug-induced readthrough of premature stop codons leads to the stabilization of laminin alpha2 chain mRNA in CMD myotubes.
    J Gene Med, 2008 Feb; 10 (2):217-24.
  • Six I, Hermida JS, Huang H, Gouas L, Fressart V, Benammar N, Hainque B, Denjoy I, Chahine M, Guicheney P.
    The occurrence of Brugada syndrome and isolated cardiac conductive disease in the same family could be due to a single SCN5A mutation or to the accidental association of both diseases.
    Europace. 2008 Jan;10(1):79-85.
  • Fligny C, Fromes Y, Bonnin P, Darmon M, Bayard E, Launay JM, Cot F, Mallet J, Vodjdani G.
    Maternal serotonin influences cardiac function in adult offspring.
    FASEB J, 2008 Feb 11, [Epub ahead of print]
  • Kaplan JC, Chelly J, Garcia L.
    Un saut symbolique mais encourageant dans le traitement de la myopathie de Duchenne.
    Med Sci (Paris), 2008 Feb; 24 (2):215-218.
  • Moresi V, Pristera A, Scicchitano BM, Molinaro M, Teodori L, Sassoon D, Adamo S, Coletti D.
    TNF Inhibition of Skeletal Muscle Regeneration is Mediated by a Caspase Dependent Stem Cell Response.
    Stem Cells, 2008 Feb 7 [Epub ahead of print].
  • Rederstorff M, Allamand V, Guicheney P, Gartioux C, Richard P, Chaigne D, Krol A, Lescure A.
    Ex vivo correction of selenoprotein N deficiency in rigid spine muscular dystrophy caused by a mutation in the selenocysteine codon.
    Nucleic Acids Res. 2008 Jan;36(1):237-44.

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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.

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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.

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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

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Books

  • A Short Guide to the Human Genome
    By Stewart Scherer
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

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  • Multiplicity Yours - Cloning, Stem Cell Research, and Regenerative Medicine
    By Hwa A Lim
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.

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Recruitement

  • Physiotherapist/ Trials Coordinator - Newcastle University, UK.
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)
  • Research Assistant : Social and ethical aspects of research in Neuromuscular disorders (50%) -Newcastle University, UK.
For further information please contact: Dr Simon Woods, 0191 243 0783, simon.woods(a)ncl.ac.uk.
  • Research Assistant and Technical Assistant - University of Oxford, Department of Physiology, Anatomy and Genetics
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
  • Postdoctoral positions
    Department of Neurobiology and Genetics at IGBMC-Strasbourg
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

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   Subscription

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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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