Improvements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study. - Publication

Publication

Improvements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study.

Journal Paper/Review - Jan 1, 2023

Pechmann Astrid, Behrens Max, Dörnbrack Katharina, Tassoni Adrian, Wenzel Franziska, Stein Sabine, Vogt Sibylle, Zöller Daniela, Bernert Günther, Hagenacker Tim, Schara-Schmidt Ulrike, Walter M C, Steinbach Meike, Blaschek Astrid, Baumann Matthias, Baumgartner Manuela, Becker Benedikt, Flotats-Bastardas Marina, Friese Johannes, Günther René, Hahn Andreas, Küpper Hanna, Johannsen Jessika, Kamm Christoph, Koch Jan Christoph, Köhler Cornelia, Kölbel Heike, Kolzter Kirsten, von Moers Arpad, Naegel Steffen, Neuwirth Christoph, Petri Susanne, Roediger Annekathrin, Schimmel Mareike, Schrank Bertold, Schreiber Gudrun, Smitka Martin, Stadler Christian, Steiner Elisabeth, Stögmann Eva, Trollmann Regina, Türk Matthias, Weiler Markus, Stoltenburg Corinna, Willichowsky Ekkehard, Zeller Daniel, Ziegler Andreas, Lochmüller Hanns, Kirschner Janbernd, SMArtCARE study group

Citation

Pechmann A, Behrens M, Dörnbrack K, Tassoni A, Wenzel F, Stein S, Vogt S, Zöller D, Bernert G, Hagenacker T, Schara-Schmidt U, Walter M, Steinbach M, Blaschek A, Baumann M, Baumgartner M, Becker B, Flotats-Bastardas M, Friese J, Günther R, Hahn A, Küpper H, Johannsen J, Kamm C, Koch J, Köhler C, Kölbel H, Kolzter K, von Moers A, Naegel S, Neuwirth C, Petri S, Roediger A, Schimmel M, Schrank B, Schreiber G, Smitka M, Stadler C, Steiner E, Stögmann E, Trollmann R, Türk M, Weiler M, Stoltenburg C, Willichowsky E, Zeller D, Ziegler A, Lochmüller H, Kirschner J, SMArtCARE study group. Improvements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study. J Neuromuscul Dis 2023; 10:29-40.

Type

Journal Paper/Review (English)

Journal

J Neuromuscul Dis 2023; 10

Publication Date

Jan 1, 2023

Issn Electronic

2214-3602

Pages

29-40

Brief description/objective

Disease progression in patients with spinal muscular atrophy (SMA) has changed dramatically within the past years due to the approval of three different disease-modifying treatments. Nusinersen was the first drug to be approved for the treatment of SMA patients. Clinical trials provided data from infants with SMA type 1 and children with SMA type 2, but there is still insufficient evidence and only scarcely reported long-term experience for nusinersen treatment in ambulant patients. Here, we report data from the SMArtCARE registry of ambulant patients under nusinersen treatment with a follow-up period of up to 38 months.