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Multifocal Noninvasive Magnetic Stimulation of the Primary Motor Cortex in Type 1 Myotonic Dystrophy - A Proof of Concept Pilot Study.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 963, doi. 10.3233/JND-210690
By:
- Greene, Ericka;
- Thonhoff, Jason;
- John, Blessy S.;
- Rosenfield, David B.;
- Helekar, Santosh A.;
- Appel, Stanley H.
Publication type:
Article
The Minimal Clinical Important Difference (MCID) in Annual Rate of Change of Timed Function Tests in Boys with DMD.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 939, doi. 10.3233/JND-210646
By:
- Duong, Tina;
- Canbek, Jennifer;
- Birkmeier, Marisa;
- Nelson, Leslie;
- Siener, Catherine;
- Fernandez-Fernandez, Alicia;
- Henricson, Erik;
- McDonald, Craig M.;
- Gordish-Dressman, Heather
Publication type:
Article
Subcutaneous Immunoglobulin Therapy with IgPro20 in Patients with Stiff Person Syndrome and Primary Immunodeficiency Disease.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 973, doi. 10.3233/JND-200616
By:
- Francis, Olivia L.;
- Joshi, Avni;
- Prince, Ty;
- Krishnaswamy, Guha;
- Patel, Niraj C.
Publication type:
Article
How Patient Organizations Can Drive FAIR Data Efforts to Facilitate Research and Health Care: A Report of the Virtual Second International Meeting on Duchenne Data Sharing, March 3, 2021.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 1097, doi. 10.3233/JND-210721
By:
- van Lin, Nawel;
- Paliouras, Georgios;
- Vroom, Elizabeth;
- 't Hoen, Peter A. C.;
- Roos, Marco
Publication type:
Article
Inflammatory Myositis Secondary to Anti-Retroviral Therapy in a Child; Case Report and Review of the Literature.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 1089, doi. 10.3233/JND-210669
By:
- Monaghan, Marie;
- Loh, Charlotte;
- Jones, Stephen;
- Oware, Agyepong;
- Urankar, Kathryn;
- Roderick, Marion;
- Majumdar, Anirban
Publication type:
Article
Healthcare Utilisation and Satisfaction with Care in Patients with Amyotrophic Lateral Sclerosis - An Observational Study.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 1079, doi. 10.3233/JND-210687
By:
- Kierkegaard, Marie;
- Gottberg, Kristina;
- Johansson, Sverker;
- Littorin, Susanne;
- Sandstedt, Petter;
- Ytterberg, Charlotte;
- Holmqvist, Lotta Widén
Publication type:
Article
Coping in Children and Adolescents with a Genetic Muscle Disorder - Findings from a Population-Based Study.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 1069, doi. 10.3233/JND-200564
By:
- Jones, Kelly M.;
- Anand, Aaron;
- Bright, Charmaine;
- O'Grady, Gina;
- Rodrigues, Miriam J.;
- Ranta, Annemare;
- Roxburgh, Richard H.;
- Theadom, Alice
Publication type:
Article
Have Duchenne Muscular Dystrophy Patients an Increased Cancer Risk?
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 1063, doi. 10.3233/JND-210676
By:
- Vita, Gian Luca;
- Politano, Luisa;
- Berardinelli, Angela;
- Vita, Giuseppe
Publication type:
Article
Estimation of the Quality-of-Life Impact of X-Linked Myotubular Myopathy.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 1047, doi. 10.3233/JND-210686
By:
- Lloyd, Andrew;
- Aggio, Daniel;
- Slocomb, Ted L.;
- Jun Lee;
- Beggs, Alan H.;
- Bilder, Deborah A.
Publication type:
Article
Clinical Outcome Evaluations and CBT Response Prediction in Myotonic Dystrophy.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 1031, doi. 10.3233/JND-210634
By:
- van As, Daniël;
- Okkersen, Kees;
- Bassez, Guillaume;
- Schoser, Benedikt;
- Lochmüller, Hanns;
- Glennon, Jeffrey C.;
- Knoop, Hans;
- van Engelen, Baziel G. M.;
- 't Hoen, Peter A. C.
Publication type:
Article
The Rare Knowledge Mining Methodological Framework for the Development of Practice Guidelines and Knowledge Translation Tools for Rare Diseases.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 1017, doi. 10.3233/JND-200536
By:
- Gagnon, C.;
- Fortin, J.;
- Lamontagne, M. E.;
- Plourde, A.
Publication type:
Article
What Happened with Muscle Force, Dynamic Stability And Falls? A 10-Year Longitudinal Follow-Up in Adults with Myotonic Dystrophy Type 1.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 1007, doi. 10.3233/JND-200521
By:
- Hammarén, Elisabet;
- Kollén, Lena
Publication type:
Article
COVID-19 in a Cohort of Patients with Congenital Myasthenic Syndrome.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 1003, doi. 10.3233/JND-210699
By:
- Alabaf, Setareh;
- O'Connell, Karen;
- Ramdas, Sithara;
- Beeson, David;
- Palace, Jacqueline
Publication type:
Article
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 989, doi. 10.3233/JND-210643
By:
- McDonald, Craig M.;
- Shieh, Perry B.;
- Abdel-Hamid, Hoda Z.;
- Connolly, Anne M.;
- Ciafaloni, Emma;
- Wagner, Kathryn R.;
- Goemans, Nathalie;
- Mercuri, Eugenio;
- Khan, Navid;
- Koenig, Erica;
- Malhotra, Jyoti;
- Wenfei Zhang;
- Baoguang Han;
- Mendell, Jerry R.
Publication type:
Article
Quantitative Evaluation of Upright Posture by x-Ray and 3D Stereophotogrammetry with a New Marker Set Protocol in Late Onset Pompe Disease.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 979, doi. 10.3233/JND-210663
By:
- De Blasiis, Paolo;
- Fullin, Allegra;
- Sansone, Mario;
- Del Viscovo, Luca;
- Napolitano, Filomena;
- Terracciano, Chiara;
- Lus, Giacomo;
- Melone, Mariarosa Anna Beatrice;
- Sampaolo, Simone
Publication type:
Article
Compliance to DMD Care Considerations in the Netherlands.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 927, doi. 10.3233/JND-210670
By:
- Heutinck, Lotte;
- Houwen-van Opstal, Saskia L. S.;
- Krom, Yvonne D.;
- Niks, Erik H.;
- Verschuuren, Jan J. G. M.;
- Jansen, Merel;
- de Groot, Imelda J. M.
Publication type:
Article
Diffusion Tensor Imaging Shows Differences Between Myotonic Dystrophy Type 1 and Type 2.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 949, doi. 10.3233/JND-210660
By:
- Rehmann, R.;
- Schneider-Gold, C.;
- Froeling, M.;
- Güttsches, A. K.;
- Rohm, M.;
- Forsting, J.;
- Vorgerd, M.;
- Schlaffke, L.
Publication type:
Article
Adult North Star Network (ANSN): Consensus Guideline For The Standard Of Care Of Adults With Duchenne Muscular Dystrophy.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 899, doi. 10.3233/JND-200609
By:
- Quinlivan, R.;
- Messer, B.;
- Murphy, P.;
- Astin, R.;
- Mukherjee, R.;
- Khan, J.;
- Emmanuel, A.;
- Wong, S. C.;
- Kulshresha, R.;
- Willis, T.;
- Pattni, J.;
- Willis, D.;
- Morgan, A.;
- Savvatis, K.;
- Keen, R.;
- Bourke, J.;
- Bettolo, C. Marini;
- Hewamadduma, C.
Publication type:
Article
Targeted Therapies for Leigh Syndrome: Systematic Review and Steps Towards a 'Treatabolome'.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 885, doi. 10.3233/JND-210715
By:
- May Yung Tiet;
- Zhiyuan Lin;
- Fei Gao;
- Jennings, Matthew James;
- Horvath, Rita
Publication type:
Article
Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases.
Published in:
Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 869, doi. 10.3233/JND-200560
By:
- Yoshitsugu Aoki;
- Wood, Matthew J. A.
Publication type:
Article

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