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 -