List of abbreviations
ALS Amyotrophic lateral sclerosis
ALSFRS-R Revised Amyotrophic Laterals Sclerosis Functional Rating Scale
BMD Becker muscular dystrophy
CI Confidence interval
CMT Charcot-Marie-Tooth disease
COI Cost of illness
DMD Duchenne muscular dystrophy
EQ-VAS Self-rated health on a visual analogue scale (0-100)
HRQoL Health-related quality of life
MHH Hannover Medical School
NMD Neuromuscular disorders
SMA Spinal muscular atrophy
Ethics approval and consent to participate
Ethical approval for this study was obtained from the institutional ethics board of MHH (no. 7922_BO_K_2018). All study participants gave their written consent to participate in this study.
Consent for publication
All study participants gave their written consent to the publication of anonymous (cohort) data.
Availability of data and materials
The datasets used and/or analysed during the current study are available from the corresponding author on reasonable request.
Competing interests
The authors declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article:
ES reports no conflicts of interest.
AO has received honoraria as a speaker/consultant from the German Neuromuscular Society “Deutsche Gesellschaft fuer Muskelkranke (DGM e.V.)” and Biogen GmbH. She has received academic research support by PRACTIS – Clinician Scientist Program of Hannover Medical School, funded by the German Research Foundation (DFG, ME 3696/3-1).
LHM reports no conflicts of interest.
SP has received grants by the German Neuromuscular Society (Deutsche Gesellschaft fuer Muskelkranke (DGM e.V.)), the Federal Ministry of Education and Research (BMBF), the German Israeli Foundation (GIF) for Scientific Research and Development and the EU Joint Programme for Neurodegenerative Disease Research (JPND). She has received speaking honoraria from Desitin Pharma, Biogen GmbH, Novartis and TEVA as well as fees for consulting from Biogen GmbH, Roche Pharma, Cytokinetics Inc.
OSK has received honoraria as a speaker/consultant and/or funding for travel expenses from the German Neuromuscular Society (Deutsche Gesellschaft fuer Muskelkranke (DGM e.V.)), Novartis, Biogen GmbH and the Jain Foundation. She has received academic research support by the Ludwig-Maximilians-University of Munich, FoeFoLe program, Reg.-No. 747, 11/11-05/13, and the Hannover Medical School Young Faculty Program, 2018-2020. She has received research support from the Friedrich-Baur-GmbH, Burgkunstadt 12/13-12/15 and the German Neuromuscular Society “Deutsche Gesellschaft fuer Muskelkranke (DGM e.V.), 2019-2020.
Funding
Deutsche Forschungsgemeinschaft PRACTIS (Program of Hannover Medical School for Clinician Scientists) (grant to A. Osmanovic). German Neuromuscular Society (Deutsche Gesellschaft fuer Muskelkranke (DGM e.V.)), Federal Ministry of Education and Research, German Israeli Foundation for Scientific Research and Development, EU Joint Programme for Neurodegenerative Disease Research (grants to S. Petri). Academic research support by MHH Young Faculty Program, 2018-2020, as well as research support by the German Neuromuscular Society (Deutsche Gesellschaft fuer Muskelkranke (DGM e.V.)), 2019-2020 (grants to O. Schreiber-Katz).
Author contributions
ES played a major role in the acquisition, analysis and interpretation of data, drafted the manuscript and set the figures and tables in this manuscript.
AO supported the patient recruitment and revised the manuscript for intellectual content.
LHM supported the patient recruitment and revised the manuscript for intellectual content.
SP took part in the creation of the study concept and design and revised the manuscript for intellectual content.
OSK played a major role in the creation of the study concept and design, was the study supervisor, designed the questionnaire with input from the other authors, took part in the acquisition, analysis and interpretation of data and revised the manuscript.
Acknowledgements
The authors thank Prof. Dr. rer. pol. Christian Krauth, Institute for Epidemiology, Social Medicine and Health Systems Research, Hannover Medical School, for his input in questionnaire design. We thank Sebastian Häckl, M. Sc. Public Health, Institute for Biostatistics, Hannover Medical School, for advice during the statistical analysis process, and James Mesiti for his insight regarding the language and expression. We also thank the German Neuromuscular Society (Deutsche Gesellschaft fuer Muskelkranke (DGM e.V.)) for funding the study. Most importantly, we thank all study participants and their caregivers.