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Volume 5, Number 2 / 2018 Now Available Online

Review Article
Current Classification and Management of Inflammatory MyopathiesOpen Access
Schmidt, Jens

Article Commentary
Position Statement: Sharing of Clinical Research Data in Spinal Muscular Atrophy to Accelerate Research and Improve Outcomes for PatientsOpen Access
Lochmüller, Hanns | Evans, David | Farwell, Wildon | Finkel, Richard | Goemans, Nathalie | de Lemus, Mencia | Matyushenko, Vitaliy | Muntoni, Francesco | Ouillade, Marie-Christine | Schwersenz, Inge | Wilson, Petra

Research Articles
Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in GermanyOpen Access
Pechmann, Astrid | Langer, Thorsten | Schorling, David | Stein, Sabine | Vogt, Sibylle | Schara, Ulrike | Kölbel, Heike | Schwartz, Oliver | Hahn, Andreas | Giese, Kerstin | Johannsen, Jessika | Denecke, Jonas | Weiß, Claudia | Theophil, Manuela | Kirschner, Janbernd

Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn ScreeningOpen Access
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

Evaluator Training and Reliability for SMA Global Nusinersen Trials
Glanzman, Allan M. | Mazzone, Elena S. | Young, Sally Dunaway | Gee, Richard | Rose, Kristy | Mayhew, Anna | Nelson, Leslie | Yun, Chris | Alexander, Katie | Darras, Basil T. | Zolkipli-Cunningham, Zarazuela | Tennekoon, Gihan | Day, John W. | Finkel, Richard S. | Mercuri, Eugenio | De Vivo, Darryl C. | Baldwin, Ron | Bishop, Kathie M. | Montes, Jacqueline

An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA membersOpen Access
Belter, Lisa | Cook, Suzanne F. | Crawford, Thomas O. | Jarecki, Jill | Jones, Cynthia C. | Kissel, John T. | Schroth, Mary | Hobby, Kenneth

Determination of qPCR Reference Genes Suitable for Normalizing Gene Expression in a Canine Model of Duchenne Muscular Dystrophy
Hildyard, John C.W. | Taylor-Brown, Frances | Massey, Claire | Wells, D.J. | Piercy, Richard J.

Characteristics of Japanese Patients with Becker Muscular Dystrophy and Intermediate Muscular Dystrophy in a Japanese National Registry of Muscular Dystrophy (Remudy): Heterogeneity and Clinical VariationOpen Access
Mori-Yoshimura, Madoka | Mitsuhashi, Satomi | Nakamura, Harumasa | Komaki, Hirofumi | Goto, Kanako | Yonemoto, Naohiro | Takeuchi, Fumi | Hayashi, Yukiko K. | Murata, Miho | Takahashi, Yuji | Nishino, Ichizo | Takeda, Shin’ichi | Kimura, En

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Most Read JND Articles in February to May 2018

Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in GermanyOpen Access (Research Article in Vol.5, Iss.2, 2018)
Pechmann, Astrid | Langer, Thorsten | Schorling, David | Stein, Sabine | Vogt, Sibylle | Schara, Ulrike | Kölbel, Heike | Schwartz, Oliver | Hahn, Andreas | Giese, Kerstin | Johannsen, Jessika | Denecke, Jonas | Weiß, Claudia | Theophil, Manuela | Kirschner, Janbernd

Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural HistoryOpen Access (Research Article in Vol.5, Iss.1, 2018)
Kinane, T. Bernard | Mayer, Oscar H. | Duda, Petra W. | Lowes, Linda P. | Moody, Stephanie L. | Mendell, Jerry R.

Anti-HMGCR MyopathyOpen Access (Review Article in Vol.5, Iss.1, 2018)
Mohassel, Payam | Mammen, Andrew L.

Myotonic Dystrophy Type 2: An Update on Clinical Aspects, Genetic and Pathomolecular MechanismOpen Access (Research Article in Vol.2, Iss.2, 2015)
Meola, Giovanni | Cardani, Rosanna

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

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TREAT-NMD News

ERN-EURO-NMD & TREAT-NMD Translational Research Summer School

July 2–6, 2018 | Newcastle University, UK

This year sees the European Reference Network for Neuromuscular Diseases (EURO-NMD) in association with TREAT-NMD deliver a ground-breaking Summer School. The unique, week-long course follows a potential therapy’s complete journey from ‘bench to bedside’. Attendees who successfully complete this course will leave with a comprehensive ‘second to none’ understanding of the processes, principles and concepts involved, as well as a distinct advantage when working in the area of clinical trials. Registration is €500 and there is still time to apply via the website.

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TREAT-NMD's 6th International Conference 2019

Location: Leiden, the Netherlands

The TREAT-NMD Alliance is pleased to announce that its next international conference will be held in Leiden, the Netherlands in 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, including dates and venue, will be released via the conference website in due course.

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Upcoming Meetings

16th ENCALS Meeting

June 20–22, 2018 | Oxford, UK

The 16th European Network to Cure ALS (ENCALS) meeting will be held in Oxford this month and will welcome scientists and clinicians who are working to find a cure for ALS/MND. Members of the JND Editorial Board will also be in attendance. Discover more about the event at the ENCALS website here.

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15th International Congress on Neuromuscular Disease

July 6–10, 2018 | Vienna, Austria

The International Congress on Neuromuscular Disease (ICNMD) will have a program presenting a wide spectrum of neuromuscular diseases from the perspectives of advances in research, diagnosis and treatment. The event in Vienna in July will provide an opportunity to obtain new perspectives on neuromuscular diseases by exchanging knowledge, expertise and ideas with esteemed colleagues. Discover more at the event website here.

JND will have a table at INCMD: come see us there and consider submitting your paper, or pick up a free copy of the journal.

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Indexed in: PubMed/MEDLINE Scopus

Submit your Paper

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-print) and an affordable Open Access option (US$1450 / €1250). JND is fully compliant with all mandates by major funders.

Submit your manuscript online via MsTracker
View detailed Instructions to Authors
Aims and Scope

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