Clinical evaluation and monitoring of patients with spinal muscular atrophy




The natural history of SMA changed over the last decade due to changes in care and the development of targeted treatments for SMA.

Targeted treatments for SMA have been developed in preclinical and clinical studies. Among these, Nusinersen is the first drug to be approved in the United States and in Europe since 2016 and 2017 for the treatment of SMA. The approval of nusinersen for all types of SMA is based on well-controlled clinical trial data of a small subgroup of pediatric SMA patients. Thus, a systematical data capture is crucial to evaluate outcomes and the effect of treatment after prolonged treatment periods and in a broader spectrum of patients.

In Germany, there is already an existing registry for patients with spinal muscular atrophy. Please, indicate this opportunity to your patients (www.sma-register.de).

SMArtCARE is a joint initiative of neurologists, child neurologists, and patients’ organizations. The aim of SMArtCARE is to collect longitudinal “real-world data” on all available SMA patients independent of their actual treatment regime as disease-specific SMA registry. For this purpose, an online platform for SMA patients will be provided seen by health-care providers in German-speaking countries. Data are collected during routine patient visits and items for data collection are aligned with the international consensus for SMA registries.

Biogen provides financial support for this registry. In the future, further support from other companies is conceivable to secure the long-term sustainability of the registry. However, the contractual basis has been regulated in such a way that data collection and interpretation are independent of pharmaceutical companies.

We have already developed recommendations for the clinical evaluation of patients with SMA and provide corresponding documentation sheets in the download area. Compensation is provided for the documentation efforts of the participating centers and is contractually guaranteed. If you are interested in participating in the SMArtCARE data collection, please contact us: SMArtCARE@uniklinik-freiburg.de. The data would first be collected at your center. If the corresponding ethical approval and consent of the patients are available, the transfer to the central data collection is possible later.

SMArtCARE is a network for clinical research in spinal muscular atrophy. If you have your own research ideas or would like to validate new evaluation tools, please contact us. You may be able to use the infrastructure of SMArtCARE for your project, thus avoiding unnecessary extra work.

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SMArtCARE Steering Committee

Prof. Dr. Günther Bernert
Priv.-Doz. Dr. Tim Hagenacker
Prof. Dr. Jan Kirschner
Prof. Dr. Hanns Lochmüller
Prof. Dr. Wolfgang Müller-Felber
Dr. Astrid Pechmann
Prof. Dr. Ulrike Schara
Dr. Inge Schwersenz (Patientenvertretung)
Prof. Dr. Maggie Walter

Workshops for the physiotherapeutic assessments

Workshops with detailed training on the motor function tests are offered at regular intervals. The events are carried out with the support of Biogen. If you have any questions, please contact us: SMArtCARE@uniklinik-freiburg.de.

Dates 2020Key TopicLocation
12./13. March (workshop cancelled)CHOP INTEND, HFMSE
(newcomer course)
Frankfurt
07./08. May (workshop cancelled)HFMSE, RULM, 6MWT
(advanced training)
Frankfurt
19./20. November (workshop cancelled)All assessments
(newcomer course)
Frankfurt
   
New: 12. November
16:00 - 18:00 CET
Various Topics - will be announced soon,
overview of recommendations for the evaluation of patients in SMA
Webinar/Online
New: 16. November
16:00 - 18:30 CET
CHOP and HFMSE advanced training,
overview of recommendations for the evaluation of patients in SMA
Webinar/Online
New: 23. November
16:00 - 18:30 CET
RULM and 6 MWT advanced training,
overview of recommendations for the evaluation of patients in SMA
Webinar/Online
   
Dates 2019Location
14./15. MarchCHOP INTEND
(newcomer course)
Frankfurt
23./24. MayHFMSE, RULM, 6MWT
(newcomer course)
Frankfurt
04./05. JulyCHOP INTEND, HFMSE
(advanced training)
Frankfurt
28./29. NovemberAll assessments
(advanced training)
Frankfurt
   
Dates 2018 Location
22./23. February Berlin
19./20. April Frankfurt
7./8. June Frankfurt
15./16. November Berlin
   
Dates 2017 Location
March Frankfurt
May Frankfurt
October Frankfurt
November Frankfurt

Downloads

SMArtCARE Flyer
Recommendations for the evaluation of patients with SMA
CRFs and Manuals (please ask for login and password: SMArtCARE@uniklinik-freiburg.de)

Publications

Treatment with Nusinersen – Challenges Regarding the Indication for Children with SMA Type 1
     (Pechmann A et al., Journal of neuromuscular diseases, 2020)
Zur Gentherapie der Spinalen Muskelatrophie mit Onasemnogene Abeparvovec. Stellungnahme der Gesellschaft für Neuropädiatrie
     (Kirschner J et al., Monatsschrift Kinderheilkunde, 2020)
Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study
     (Hagenacker T et al., The Lancet. Neurology, 2020)
SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy
     (Pechmann A et al., Orphanet Journal of Rare Diseases, 2019)
Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany
     (Pechmann A et al., Journal of Neuromuscular Diseases, 2018)
Therapie der 5q-assoziierten Spinalen Muskelatrophie mit Nusinersen – erste Erfahrungen
     (Giese K et al., Neuropädiatrie in Klinik und Praxis, 2018, Max Schmidt-Römhild GmbH & Co. KG)
International consensus on the therapie of SMA (Neuromuscular Disorders 2018): Part 1 und Part 2

Important Links

SMA Patient Registry
Initiative SMA
Deutsche Muskelstiftung
Deutsche Gesellschaft für Muskelkranke

Map of the participating centres



Contact

Principal Investigator: Dr. Astrid Pechmann

Department of Neuropediatrics and Muscle Disorders
Medical Center- University of Freiburg

If you have any questions, please write to us: SMArtCARE@uniklinik-freiburg.de
For questions on physiotherapie: smartcare-physio@uniklinik-freiburg.de

Support

For technical support please contact us via email: SMArtCARE-support@uniklinik-freiburg.de
or by phone: +49-761-270-43211



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