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Volume 5, Issue 4 / 2018 Now Available Online

Review Articles
Centronuclear Myopathies Under Attack: A Pethora of Therapeutic TargetsOpen Access
Tasfaout, Hichem | Cowling, Belinda S. | Laporte, Jocelyn

“Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the ClinicOpen Access
Gordish-Dressman, Heather | Willmann, Raffaella | Dalle Pazze, Laura | Kreibich, Arati | van Putten, Maaike | Heydemann, Ahlke | Bogdanik, Laurent | Lutz, Cathleen | Davies, Kay | et al.

Research Articles
Home-Based Monitoring of Pulmonary Function in Patients with Duchenne Muscular DystrophOpen Access
Buyse, Gunnar M. | Rummey, Christian | Meier, Thomas | Leinonen, Mika | Voit, Thomas | McDonald, Craig M. | Mayer, Oscar H.

Respiratory Assessment of ALS Patients: A Nationwide Survey of Current Dutch Practice
Tilanus, T.B.M. | Groothuis, J.T. | ten Broek-Pastoor, J.M.C. | Doorduin, J. | van Engelen, B.G.M. | Kampelmacher, M.J. | Raaphorst, J.

Disability and Contextual Factors in Patients with Amyotrophic Lateral Sclerosis – A Three-Year Observational StudyOpen Access
Sandstedt, Petter | Littorin, Susanne | Johansson, Sverker | Gottberg, Kristina | Ytterberg, Charlotte | Kierkegaard, Marie

How to Interpret Abnormal Findings of Spirometry and Manometry in Myotonic Dystrophies?
Babačić, Haris | Goldina, Olga | Stahl, Kristina | Montagnese, Federica | Jurinović, Vindi | Schoser, Benedikt | Wenninger, Stephan

Myotonic Dystrophy Type 2 – Data from the Serbian Registry
Bozovic, Ivo | Peric, Stojan | Pesovic, Jovan | Bjelica, Bogdan | Brkusanin, Milos | Basta, Ivana | Bozic, Marija | Sencanic, Ivan | Marjanovic, Ana | Brankovic, Marija | Savic-Pavicevic, Dusanka | Rakocevic-Stojanovic, Vidosava

Low Prevalence Estimates of Late-Onset Glycogen Storage Disease Type II in French-Speaking Belgium are not Due to Missed Diagnoses
Remiche, Gauthier | Lukacs, Zoltan | Kasper, David C. | Abramowicz, Marc | Pandolfo, Massimo

Diagnostic Accuracy of Phenotype Classification in Duchenne and Becker Muscular Dystrophy Using Medical Record Data
Andrews, Jennifer G. | Lamb, Molly M. | Conway, Kristin | Street, Natalie | Westfield, Christina | Ciafaloni, Emma | Matthews, Dennis | Cunniff, Christopher | Pandya, Shree | Fox, Deborah J. | MD STARnet

Bone Health and Endocrine Care of Boys with Duchenne Muscular Dystrophy: Data from the MD STARnet
Weber, David R. | Thomas, Shiny | Erickson, Stephen W. | Fox, Deborah | Oleszek, Joyce | Pandya, Shree | Venkatesh, Yedatore | Westfield, Christina | Ciafaloni, Emma | MD STARnet

Motor Function Test Reliability During the NeuroNEXT Spinal Muscular Atrophy Infant Biomarker Study
Krosschell, Kristin J. | Bosch, Michael | Nelson, Leslie | Duong, Tina | Lowes, Linda P. | Alfano, Lindsay N. | Benjamin, Danielle | Carry, Terri B. | Devine, Ginger | Kelley, Carolyn | et al.

Meeting Reports
Report of the Third Outcome Measures in Myotonic Dystrophy Type 1 International Workshop – Paris, France Openly Available
Gagnon, Cynthia | Heatwole, Chad | Hébert, Luc J. | Hogrel, Jean-Yves | Laberge, Luc | Leone, Mario | Meola, Giovanni | Richer, Louis | Sansone, Valeria | Kierkegaard, Marie

Report on the 4th Ottawa International Conference on Neuromuscular Disease and Biology – Ottawa, CanadaOpenly Available
Chardon, Jodi Warman | Jasmin, Bernard J. | Kothary, Rashmi | Parks, Robin J.

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Most Read JND Articles in October 2018

15th International Congress on Neuromuscular Diseases, July 6 - 10, 2018 Vienna, AustriaOpen Access (Meeting Abstracts in Vol.5, Iss.s1, 2018)
Various Authors

New Pathways and Therapeutic Targets in Autoimmune Myasthenia GravisOpen Access (Review Article in Vol.5, Iss.3, 2018)
Behin, Anthony | Le Panse, Rozen

Current Classification and Management of Inflammatory MyopathiesOpen Access (Review Article in Vol.5, Iss.2, 2018)
Schmidt, Jens

Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn ScreeningOpen Access (Research Article in Vol.5, Iss.2, 2018)
Glascock, Jacqueline | Sampson, Jacinda | Haidet-Phillips, Amanda | Connolly, Anne | Darras, Basil | Day, John | Finkel, Richard | Howell, R. Rodney | Klinger, Katherine | Kuntz, Nancy | Prior, Thomas | Shieh, Perry B. | Crawford, Thomas O. | Kerr, Douglas | Jarecki, Jill

“Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the ClinicOpen Access (Review Article in Vol.5, Iss.4, 2018)
Gordish-Dressman, Heather | Willmann, Raffaella | Dalle Pazze, Laura | Kreibich, Arati | van Putten, Maaike | Heydemann, Ahlke | Bogdanik, Laurent | Lutz, Cathleen | Davies, Kay | et al.

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New DMD Family Guide

Using updated recommendations, TREAT-NMD has worked together with the Muscular Dystrophy Association USA, Parent Project Muscular Dystrophy, and the World Duchenne Organisation UPPMD to produce a current and comprehensive Duchenne Guide for Families ensuring that the very latest in care standards are accessible to all people living with Duchenne. The new guide was launched on 7 September, to coincide with Duchenne Awareness Day. You can find more information about the publication on the TREAT-NMD website.

SMA Family Guide and Expert Masterclass

Following the publication of the SMA Care Recommendations in January 2018, TREAT-NMD has been working with MDUK, SMASupportUK, SMA Europe and Cure SMA to develop a medical guide for families, known as the SMA Family Guide. The support and expertise of the neuromuscular community is called upon to translate this guide. If you can help, please get in touch.

Also in the field of SMA, the first SMA Expert Masterclass will be co-chaired by Professor Eugenio Mercuri and Professor Nathalie Goemans on 19–20 November in Rome, Italy. This event will provide a platform for health care providers to share ideas, challenges and successes in delivering optimal care to people with SMA. The masterclass is suitable for all health care providers who may encounter and provide care to patients with SMA. For further details, see here.

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Upcoming Events

29th International Symposium on ALS/MND

7–9 December 2018 | Glasgow, UK

The International Symposium on ALS/MND is the largest medical and scientific conference specific to MND/ALS and is the premier forum for presentation and discussion of new biomedical research findings and clinical management of the disease. Register before 20 November and you can save £290 compared to the onsite registration fee. For more details, visit the website.


TREAT-NMD's 6th International Conference 2019

9–11 December 2019 | Leiden, the Netherlands // Save the Date!

The TREAT-NMD Alliance will hold its next international conference in Leiden, the Netherlands in December 2019. The conference will provide an engaging platform for clinical specialists, academics, patients and carers, advocacy organisations and Industry professionals to share progress and lessons learned in the area of translational medicine in inherited neuromuscular diseases and plan for the delivery of future therapies to patients. Further details will be released via the conference website in due course.

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Indexed in: PubMed/MEDLINE Scopus

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As a member of our research community, we would like to invite you to contribute your own articles to the journal. JND offers contributing authors many benefits, including rigorous peer review and speedy manuscript processing, rapid online publication (pre-press) and an affordable Open Access option (€1250/US$1450). JND is fully compliant with all mandates by major funders, and Open Access are automatically uploaded to PubMed Central. More reasons to publish in JND are outlined on the journal's website!

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