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JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam

Risdiplam therapy in adults with 5q-SMA: observational study on motor function and treatment satisfaction

2024 . Bjelica, Bogdan | Wohnrade, Camilla | Cespedes, Iraima | Osmanovic, Alma | Schreiber-Katz, Olivia | Petri, Susanne

Risdiplam improves subjective swallowing quality in non-ambulatory adult patients with 5q-spinal muscular atrophy despite advanced motor impairment

2024 . Brakemeier, S. | Lipka, J. | Schlag, M. | Kleinschnitz, C. | Hagenacker, Tim

Identification of the most relevant aspects of spinal muscular atrophy (SMA) with impact on the quality of life of SMA patients and their caregivers: the PROfuture project, a qualitative study

2024 . de Lemus, Mencía | Cattinari, Maria Grazia | Pascual, Samuel I. | Medina, Julita | García, Mar | Magallón, Ana | Dumont, María | Rebollo, Pablo

Thrombospondin-4 as potential cerebrospinal fluid biomarker for therapy response in pediatric spinal muscular atrophy

2024 . Dobelmann, Vera | Roos, Andreas | Hentschel, Andreas | Della Marina, Adela | Leo, Markus | Schmitt, Linda-Isabell | Maggi, Lorenzo | Schara-Schmidt, Ulrike | Hagenacker, Tim | Ruck, Tobias | Kölbel, Heike