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
Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › fagfællebedømt
•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.
•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.
| Originalsprog | Engelsk |
|---|---|
| Tidsskrift | Neuromuscular Disorders |
| Vol/bind | 33 |
| Udgave nummer | 6 |
| Sider (fra-til) | 511-522 |
| Antal sider | 12 |
| ISSN | 0960-8966 |
| DOI | |
| Status | Udgivet - 2023 |
Bibliografisk 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.
ID: 355148633