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Volume 6, Issue 1 / 2019 Now Available Online

Review Articles
Exploration of New Contrasts, Targets, and MR Imaging and Spectroscopy Techniques for Neuromuscular DiseaseOpen Access
Strijkers, Gustav J. | Araujo, Ericky C.A. | Azzabou, Noura | Bendahan, David | Blamire, Andrew | Burakiewicz, Jedrek | Carlier, Pierre G. | Damon, Bruce | Deligianni, Xeni | Froeling, Martijn | Heerschap, Arend | Hollingsworth, Kieren G. | Hooijmans, Melissa T. | Karampinos, Dimitrios C. | et al.

Management of Adrenal Insufficiency Risk After Long-term Systemic Glucocorticoid Therapy in Duchenne Muscular Dystrophy: Clinical Practice RecommendationsOpen Access
Bowden, Sasigarn A. | Connolly, Anne M. | Kinnett, Kathi | Zeitler, Philip S.

Research Articles
Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF- κB Inhibitor, in Pediatric Patients with Duchenne Muscular DystrophyOpen Access
Finanger, Erika | Vandenborne, Krista | Finkel, Richard S. | Lee Sweeney, H. | Tennekoon, Gihan | Yum, Sabrina | Mancini, Maria | Bista, Pradeep | Nichols, Andrew | Liu, Hanlan | Fretzen, Angelika | Donovan, Joanne M.

Echographic Assessment of Diaphragmatic Function in Duchenne Muscular Dystrophy from Childhood to Adulthood
Fayssoil, Abdallah | Chaffaut, Cendrine | Ogna, Adam | Stojkovic, Tanya | Lamothe, Laure | Mompoint, Dominique | Meng, Paris | Prigent, Helene | Clair, Bernard | Behin, Anthony | Laforet, Pascal | Bassez, Guillaume | Carlier, Robert | Orlikowski, David | Amthor, Helge | Quijano Roy, Susana | Crenn, Pascal | Chevret, Sylvie | Eymard, Bruno | Lofaso, Frederic | Annane, Djillali

Increased Blood Pressure and Body Mass Index as Potential Modifiable Factors in The Progression of Myocardial Dysfunction in Duchenne Muscular Dystrophy
van de Velde, Nienke Marije | Roest, Arno Anne Willem | van Zwet, Erik Willem | Niks, Erik Harmen

Lower Extremity Functional Outcome Measures in Duchenne Muscular Dystrophy-A Delphi Survey
Senesac, Claudia R. | Lott, Donovan J. | Willcocks, Rebecca J. | Duong, Tina | Smith, Barbara K.

Pattern of Habitual Physical Exercise in Myasthenia Gravis Patients
O’Connor, Laura | Westerberg, Elisabet | Punga, Anna Rostedt

Muscle Strength and Aerobic Capacity in Patients with CIDP One Year after Participation in an Exercise Trial
Markvardsen, Lars K. | Carstens, Anne-Kathrine R. | Knak, Kirsten L. | Overgaard, Kristian | Vissing, John | Andersen, Henning

Responsiveness to Change of 5-point MRC scale, Endurance and Functional Evaluation for Assessing Myositis in Daily Clinical Practice
Landon-Cardinal, Océane | Devilliers, Hervé | Chavarot, Nathalie | Mariampillai, Kuberaka | Rigolet, Aude | Hervier, Baptiste | Allenbach, Yves | Benveniste, Olivier

Perceived Fatigue in Spinal Muscular Atrophy: A Pilot Study
Dunaway Young, Sally | Montes, Jacqueline | Kramer, Samantha S. | Podwika, Bernadette | Rao, Ashwini K. | De Vivo, Darryl C.

Perspectives on Spinraza (Nusinersen) Treatment Study: Views of Individuals and Parents of Children Diagnosed with Spinal Muscular Atrophy
Pacione, Michelle | Siskind, Carly E. | Day, John W. | Tabor, Holly K.

Reliability and Validity of Self-Report Questionnaires as Indicators of Fatigue in RYR1-Related Disorders
Kuo, Anna | Todd, Joshua J. | Witherspoon, Jessica W. | Lawal, Tokunbor A. | Elliott, Jeffery | Chrismer, Irene C. | Shelton, Monique O. | Razaqyar, Muslima S. | Jain, Minal S. | Vasavada, Ruhi | Waite, Melissa | Drinkard, Bart | Michael, Darren | Richarte, Alicia | Bönnemann, Carsten G. | Meilleur, Katherine G.

Homozygous Nonsense Mutation p.Q274X in TRIM63 (MuRF1) in a Patient with Mild Skeletal Myopathy and Cardiac Hypertrophy
Jokela, Manu | Baumann, Peter | Huovinen, Sanna | Penttilä, Sini | Udd, Bjarne

Other Content
Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification MethodologyOpen Access
Aartsma-Rus, Annemieke | Morgan, Jennifer | Lonkar, Pallavi | Neubert, Hendrik | Owens, Jane | Binks, Michael | Montolio, Marisol | Phadke, Rahul | Datson, Nicole | Van Deutekom, Judith | Morris, Glenn E. | Rao, V. Ashutosh | Hoffman, Eric P. | Muntoni, Francesco | Arechavala-Gomeza, Virginia | on behalf of the workshop participants

The Position of Neuromuscular Patients in Shared Decision Making. Report from the 235th ENMC Workshop: Milan, Italy, January 19-20, 2018Openly Available (view press release here)
Lochmüller, Hanns | Ambrosini, Anna | van Engelen, Baziel | Hansson, Mats | Tibben, Aad | Breukel, Alexandra | Sterrenburg, Ellen | Schrijvers, Guus | Meijer, Ingeborg | Padberg, George | Peay, Holly | Monaco, Lucia | Snape, Mike | Lennox, Anne | Mazzone, Elena | Bere, Nathalie | de Lemus, Mencia | Landfeldt, Erik | Willmann, Raffaella | behalf of the 235th ENMC workshop study group

Myotubular Trust - 2019 Call for Projects (Open to International Applications)Openly Available Announcement

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Most Read JND Articles in January 2019

Listing articles published only in 2018 and 2019 so you can read the most popular recent content

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

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

Diaphragm: Pathophysiology and Ultrasound Imaging in Neuromuscular DisordersOpen Access (Review Article in Vol.5, Iss.1, 2018)
Fayssoil, Abdallah | Behin, Anthony | Ogna, Adam | Mompoint, Dominique | Amthor, Helge | Clair, Bernard | Laforet, Pascal | Mansart, Arnaud | Prigent, Helene | Orlikowski, David | Stojkovic, Tanya | Vinit, Stéphane | Carlier, Robert | Eymard, Bruno | Lofaso, Frederic | Annane, Djillali

Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification MethodologyOpen Access (Meeting Report in Vol.6, Iss.1, 2019)
Aartsma-Rus, Annemieke | Morgan, Jennifer | Lonkar, Pallavi | Neubert, Hendrik | Owens, Jane | Binks, Michael | Montolio, Marisol | Phadke, Rahul | Datson, Nicole | Van Deutekom, Judith | Morris, Glenn E. | Rao, V. Ashutosh | Hoffman, Eric P. | Muntoni, Francesco | Arechavala-Gomeza, Virginia | on behalf of the workshop participants

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TREAT-NMD & EURO-NMD Translational Research Summer School

July 1–5, 2019 | Leiden, the Netherlands

The 2019 Neuromuscular Translational Summer School will be taking place from 1st to 5th July at Leiden University Medical Center in the Netherlands.

The aim of the school is to facilitate the clinical development of therapies for NMDs, to educate clinicians and researchers working in the NMD field on aspects relevant for translational therapy development and to outline and showcase how networks like EURO-NMD and TREAT-NMD facilitate therapy development. Further details and registration information are available via the EURO-NMD website.

TREAT-NMD's 6th International Conference 2019

December 9–11, 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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