Evidence map›Paper›PMID 33843694›Full record

ArticleJournal of neuromuscular diseases2021

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 and 13 more

Open access · bronzeAbstract readConference Proceedings
In one paragraph

Article in Journal of neuromuscular diseases, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
0.5field-weighted citation impact, top 32% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed, 6 citations in OpenAlex.

  1. Review
  2. Article
  3. Article
  4. Frailties and critical issues in neuromuscular diseases highlighted by SARS-CoV-2 pandemic: how many patients are still "invisible"?Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2022
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

23 authors at 17 institutions in 9 countries.

Jonathan PiniUniversité Côte d'Azur (UCA), Centre Hospitalier Universitaire de Nice, Peripheral Nervous System and Muscle Department, Rare Neuromuscular Disease Reference Center, ERN-Euro-NMD, Nice, France.
Gabriele SicilianoNeurological Clinic, Department of Clinical and Experimental Medicine, Ospedale Santa Chiara, University of Pisa, Pisa, Italy.
Pauline LahautUniversité Côte d'Azur (UCA), Centre Hospitalier Universitaire de Nice, Peripheral Nervous System and Muscle Department, Rare Neuromuscular Disease Reference Center, ERN-Euro-NMD, Nice, France.
Serge BraunAFM Telethon, Paris, France.
Sandrine Segovia-KuenyAFM Telethon, Paris, France.
Anna KolePublic Health Policy Director Rare 2030 Lead EURORDIS.
Ines HérnandoHealthcare Director EURORDIS.
Julij SelbUniversity Clinic Golnik, Golnik, Slovenia -Medical consultant Parsek, Vienna, Austria.
Erika SchirinziNeurological Clinic, Department of Clinical and Experimental Medicine, Ospedale Santa Chiara, University of Pisa, Pisa, Italy.
Tina DuongDepartment of Neurology Stanford University, Palo Alto, CA, USA.
Jean-Yves HogrelNeuromuscular Physiology and Evaluation Lab, Neuromuscular Investigation Centre, Institute of Myology, Paris, France.
José Javier Serrano OlmedoLaboratory of Bioinstrumentation and Nanomedicine, Center for Biomedical Technology, Universidad Politécnica de Madrid, Madrid, Spain.
John VissingCopenhagen Neuromuscular Center, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.
Laurent ServaisMDUK Oxford Neuromuscular Center Department of Pediatrics University of Oxford, Oxford, UK.
Dominique Vincent-GenodL'Escale, Pediatric Rehabilitation Department, Hospices Civils de Lyon, Lyon, France.
Carole VuillerotNeuron Interaction Team, NeuroMyogène Institute, Lyon University, Lyon, France.
Sylvie BannwarthDepartment of Medical Genetics, National Center for Mitochondrial Diseases, Nice University Hospital, Nice, France.
Damien EggenspielerHealthcare Program Director, Sysnav, Paris, France.
Savine VicartChannelopahies Reference Center, Service of Neuro-Myology, University Hospital Pitié-Salpêtrière, Assistance Publique-Hôpitaux de Paris, Paris, France.
Jordi Diaz-ManeraJohn Walton Muscular Dystrophy Research Center, Newcastle University, Newcastle, UK.
eNMD group
Hanns LochmüllerChildrens Hospital of Eastern Ontario Research Institute; Division of Neurology, Department of Medicine, The Ottawa Hospital; and Brain and Mind Research Institute, University of Ottawa, Ottawa, Canada.
Sabrina SacconiUniversité Côte d'Azur (UCA), Centre Hospitalier Universitaire de Nice, Peripheral Nervous System and Muscle Department, Rare Neuromuscular Disease Reference Center, ERN-Euro-NMD, Nice, France.
Université Côte d'Azur · FRAssociation Francaise contre les Myopathies · FREuropean Organisation for Rare Diseases · FRUniversity of Ottawa · CAUniversity of Pisa · ITAssistance Publique – Hôpitaux de Paris · FRHospices Civils de Lyon · FRHospital de Sant Pau · ESInstitut de Myologie · FRInstitut de Recherche sur le Cancer et le Vieillissement de Nice · FRInstitut NeuroMyoGène · FRPalo Alto University · USSysnav (France) · FRTécnicas y Servicios de Ingeniería (Spain) · ESUniversity Clinic of Pulmonary and Allergic Diseases Golnik · SIUniversity of Copenhagen · DKUniversity of Oxford · GB

Funding

Medical Research Council MC_PC_16082
6 · The paper itself

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.

Indexed as

ConsensusFranceHealth PersonnelHumansNeuromuscular DiseasesQuality of LifeTelemedicinee-HealtheNMD congressinnovationneuromuscular diseaseunmet needs

Identifiers

PMID33843694
PMCPMC8385527
OpenAlexW3146465439

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.