Works matching IS 22143599 AND DT 2021 AND VI 8 AND IP 6

Results: 20

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

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

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

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

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

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

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

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

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

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

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

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

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