Longitudinal Data Collection from Patients with Spinal Muscular Atrophy: The SMArtCARE Database

Conditions

G12.0

Schwartz O, Vill K, Pfaffenlehner M, Behrens M, Weiß C, Johannsen J, Friese J, Hahn A, Ziegler A, Illsinger S, Smitka M, von Moers A, Kölbel H, Schreiber G, Kaiser N, Wilichowski E, Flotats-Bastardas M, Husain RA, Baumann M, Köhler C, Trollmann R, Schwerin-Nagel A, Eisenkölbl A, Schimmel M, Fleger M, Kauffmann B, Wiegand G, Baumgartner M, Rauscher C, Cirak S, Gläser D, Bernert G, Hagenacker T, Goldbach S, Probst-Schendzielorz K, Lochmüller H, Müller-Felber W, Schara-Schmidt U, Walter MC, Kirschner J, Pechmann A, SMARTCARE study group.

2024-06-0127 citations

10.1001/jamapediatrics.2024.0492

SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy

Astrid Pechmann, Kirsten König, Günther Bernert, Kristina Schachtrup, Ulrike Schara et al. (13 authors)

Orphanet Journal of Rare Diseases2019-01-21135 citations

10.1186/s13023-019-0998-4

Interventions

Group 1: The aim of SMArtCARE is to collect longitudinal “real-world data” on all available SMA patients independent of their actual treatment regime as a disease-specific SMA registry. For this purpo

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: • All patients with genetically confirmed 5q SMA with residence in German-speaking regions. • Informed consent provided by patient/caregiver

Exclusion criteria

Exclusion criteria: • Further types of SMA (non 5q SMA) • Participation in an AMG trial

Design outcomes

Primary

Measure	Time frame
The aim of SMArtCARE is to collect data from all SMA patients during routine patient visits. SMArtCARE collects data on motor function as well as changes in respiratory function, nutritional status and orthopedic symptoms. To evaluate changes in motor function, standardized physiotherapeutic assessments are performed.	—

Countries

Austria, Germany, Switzerland

Contacts

Public ContactJanbernd Kirschner

Universitätsklinikum Freiburg; Klinik für Neuropädiatrie und Muskelerkrankungen

smartcare@uniklinik-freiburg.de+49 761 270 43150

Outcome results

None listed