254th ENMC international workshop. Formation of a European network to initiate a European data collection, along with development and sharing of treatment guidelines for adult SMA patients. Virtual meeting 28 – 30 January 2022

Research output: Contribution to journal › Journal article › Research › peer-review

Standard

254th ENMC international workshop. Formation of a European network to initiate a European data collection, along with development and sharing of treatment guidelines for adult SMA patients. Virtual meeting 28 – 30 January 2022. / 254th ENMC Workshop Study Group.

In: Neuromuscular Disorders, Vol. 33, No. 6, 2023, p. 511-522.

Research output: Contribution to journal › Journal article › Research › peer-review

Harvard

254th ENMC Workshop Study Group 2023, '254th ENMC international workshop. Formation of a European network to initiate a European data collection, along with development and sharing of treatment guidelines for adult SMA patients. Virtual meeting 28 – 30 January 2022', Neuromuscular Disorders, vol. 33, no. 6, pp. 511-522. https://doi.org/10.1016/j.nmd.2023.03.011

APA

254th ENMC Workshop Study Group (2023). 254th ENMC international workshop. Formation of a European network to initiate a European data collection, along with development and sharing of treatment guidelines for adult SMA patients. Virtual meeting 28 – 30 January 2022. Neuromuscular Disorders, 33(6), 511-522. https://doi.org/10.1016/j.nmd.2023.03.011

Vancouver

254th ENMC Workshop Study Group. 254th ENMC international workshop. Formation of a European network to initiate a European data collection, along with development and sharing of treatment guidelines for adult SMA patients. Virtual meeting 28 – 30 January 2022. Neuromuscular Disorders. 2023;33(6):511-522. https://doi.org/10.1016/j.nmd.2023.03.011

Author

254th ENMC Workshop Study Group. / 254th ENMC international workshop. Formation of a European network to initiate a European data collection, along with development and sharing of treatment guidelines for adult SMA patients. Virtual meeting 28 – 30 January 2022. In: Neuromuscular Disorders. 2023 ; Vol. 33, No. 6. pp. 511-522.

Bibtex

@article{5df1d50b28ca412cb28b696e23f03e92,
title = "254th ENMC international workshop. Formation of a European network to initiate a European data collection, along with development and sharing of treatment guidelines for adult SMA patients. Virtual meeting 28 – 30 January 2022",
abstract = "•Worldwide, data collections and registries, such as TREAT-NMD patient registries, iSMAC and SMArtCARE have been implemented as important tools to get more insight in the natural history of spinal muscular atrophy (SMA) in treatment-na{\"i}ve patients, especially at older ages, and patients treated with disease modifying therapy (DMT).•Specific recommendations for the transition of SMA patients from pediatric to adult care were discussed.•The group agreed on the need of a follow-up workshop on adult guidelines and development of standards of care.•Future activities of the consortium include access to treatment across Europe, implementation of new outcome measures and patient reported outcomes, (PROs), and a European data collection.",
author = "Walter, {Maggie C.} and Pascal Lafor{\^e}t and {van der Pol}, {W. Ludo} and Elena Pegoraro and Shahram Attarian and Bart Bartels and Ksenija Gorni and Nathalie Goemans and Nicole Gusset and Victoria Hodgkinson and Tim Hagenacker and Janbernd Kirschner and Andrea Klein and Anna Kostera-Pruszczyk and Hanns Lochm{\"u}ller and Chiara Marini-Bettolo and Eugenio Mercuri and Robert Muni-Lofra and Laetitia Ouillade and Rosaline Quinlivan and Constantinos Papadopoulos and H{\'e}l{\`e}ne Prigent and Emmanuelle Salort-Campana and Sansone, {Valeria A.} and Rivka Smit and Piera Smeriglio and Simone Thiele and Ben Tichler and {Van den Bergh}, Peter and Vazquez-Costa, {Juan F.} and John Vissing and {254th ENMC Workshop Study Group}",
note = "Funding Information: 29 clinical researchers from 12 different countries (Belgium, Canada, Denmark, France, Germany, Greece, Italy, Netherlands, Poland, Spain, Switzerland, UK), 2 SMA patients, one representative of a patient advocacy group and 2 industry representatives from Biogen and Roche met for a virtual workshop in the weekend of 28–30 January 2022. The Workshop aimed at the formation of a European network to initiate a European data collection, along with development and sharing of treatment guidelines for adult SMA patients. This workshop was supported by the 10 ENMC partner organisations. ",
year = "2023",
doi = "10.1016/j.nmd.2023.03.011",
language = "English",
volume = "33",
pages = "511--522",
journal = "Journal of Neuromuscular Diseases",
issn = "0960-8966",
publisher = "Elsevier",
number = "6",

}

RIS

TY - JOUR

T1 - 254th ENMC international workshop. Formation of a European network to initiate a European data collection, along with development and sharing of treatment guidelines for adult SMA patients. Virtual meeting 28 – 30 January 2022

AU - Walter, Maggie C.

AU - Laforêt, Pascal

AU - van der Pol, W. Ludo

AU - Pegoraro, Elena

AU - Attarian, Shahram

AU - Bartels, Bart

AU - Gorni, Ksenija

AU - Goemans, Nathalie

AU - Gusset, Nicole

AU - Hodgkinson, Victoria

AU - Hagenacker, Tim

AU - Kirschner, Janbernd

AU - Klein, Andrea

AU - Kostera-Pruszczyk, Anna

AU - Lochmüller, Hanns

AU - Marini-Bettolo, Chiara

AU - Mercuri, Eugenio

AU - Muni-Lofra, Robert

AU - Ouillade, Laetitia

AU - Quinlivan, Rosaline

AU - Papadopoulos, Constantinos

AU - Prigent, Hélène

AU - Salort-Campana, Emmanuelle

AU - Sansone, Valeria A.

AU - Smit, Rivka

AU - Smeriglio, Piera

AU - Thiele, Simone

AU - Tichler, Ben

AU - Van den Bergh, Peter

AU - Vazquez-Costa, Juan F.

AU - Vissing, John

AU - 254th ENMC Workshop Study Group

N1 - Funding Information: 29 clinical researchers from 12 different countries (Belgium, Canada, Denmark, France, Germany, Greece, Italy, Netherlands, Poland, Spain, Switzerland, UK), 2 SMA patients, one representative of a patient advocacy group and 2 industry representatives from Biogen and Roche met for a virtual workshop in the weekend of 28–30 January 2022. The Workshop aimed at the formation of a European network to initiate a European data collection, along with development and sharing of treatment guidelines for adult SMA patients. This workshop was supported by the 10 ENMC partner organisations.

PY - 2023

Y1 - 2023

N2 - •Worldwide, data collections and registries, such as TREAT-NMD patient registries, iSMAC and SMArtCARE have been implemented as important tools to get more insight in the natural history of spinal muscular atrophy (SMA) in treatment-naïve patients, especially at older ages, and patients treated with disease modifying therapy (DMT).•Specific recommendations for the transition of SMA patients from pediatric to adult care were discussed.•The group agreed on the need of a follow-up workshop on adult guidelines and development of standards of care.•Future activities of the consortium include access to treatment across Europe, implementation of new outcome measures and patient reported outcomes, (PROs), and a European data collection.

AB - •Worldwide, data collections and registries, such as TREAT-NMD patient registries, iSMAC and SMArtCARE have been implemented as important tools to get more insight in the natural history of spinal muscular atrophy (SMA) in treatment-naïve patients, especially at older ages, and patients treated with disease modifying therapy (DMT).•Specific recommendations for the transition of SMA patients from pediatric to adult care were discussed.•The group agreed on the need of a follow-up workshop on adult guidelines and development of standards of care.•Future activities of the consortium include access to treatment across Europe, implementation of new outcome measures and patient reported outcomes, (PROs), and a European data collection.

U2 - 10.1016/j.nmd.2023.03.011

DO - 10.1016/j.nmd.2023.03.011

M3 - Journal article

C2 - 37245491

AN - SCOPUS:85160562750

VL - 33

SP - 511

EP - 522

JO - Journal of Neuromuscular Diseases

JF - Journal of Neuromuscular Diseases

SN - 0960-8966

IS - 6

ER -

ID: 355148633