E-Health & Innovation to Overcome Barriers in Neuromuscular Diseases. Report from the 1st eNMD Congress: Nice, France, March 22-23, 2019

Jonathan Pini, Gabriele Siciliano, Pauline Lahaut, Serge Braun, Sandrine Segovia-Kueny, Anna Kole, Ines Hérnando, Julij Selb, Erika Schirinzi, Tina Duong, Jean-Yves Hogrel, José Javier Serrano Olmedo, John Vissing, Laurent Servais, Dominique Vincent-Genod, Carole Vuillerot, Sylvie Bannwarth, Damien Eggenspieler, Savine Vicart, Jordi Diaz-ManeraHanns Lochmüller, Sabrina Sacconi, eNMD group

3 Citations (Scopus)

Abstract

By definition, neuromuscular diseases are rare and fluctuating in terms of symptoms; patients are often lately diagnosed, do not have enough information to understand their condition and be proactive in their management. Usually, insufficient resources or services are available, leading to patients' social burden. From a medical perspective, the rarity of such diseases leads to the unfamiliarity of the medical staff and caregiver and an absence of consensus in disease assessment, treatment, and management. Innovations have to be developed in response to patients' and physicians' unmet needs.It is vital to improve several aspects of patients' quality of life with a better comprehension of their disease, simplify their management and follow-up, help their caregiver, and reduce the social and economic burden for living with a rare debilitating disease. Database construction regrouping patients' data and symptoms according to specific country registration on data privacy will be critical in establishing a clear consensus on neuromuscular disease treatment.Clinicians also need technological innovations to help them recognize neuromuscular diseases, find the best therapeutic approach based on medical consensus, and tools to follow patients' states regularly. Diagnosis also has to be improved by implementing automated systems to analyze a considerable amount of data, representing a significant step forward to accelerate the diagnosis and the patients' follow up. Further, the development of new tools able to precisely measure specific outcomes reliably is of the matter of importance in clinical trials to assess the efficacy of a newly developed compound.In this context, creation of an expert community is essential to communicate and share ideas. To this end, 97 clinicians, healthcare professionals, researchers, and representatives of private companies from 9 different countries met to discuss the new perspective and challenges to develop and implement innovative tools in the field of neuromuscular diseases.

Original languageEnglish
JournalJournal of neuromuscular diseases
Volume8
Issue number4
Pages (from-to)743-754
Number of pages12
DOIs
Publication statusPublished - 2021

Keywords

  • Consensus
  • France
  • Health Personnel
  • Humans
  • Neuromuscular Diseases/urine
  • Quality of Life
  • Telemedicine
  • innovation
  • eNMD congress
  • neuromuscular disease
  • unmet needs
  • e-Health

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