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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

1H-NMR-based metabolic profiling identifies non-invasive diagnostic and predictive urinary fingerprints in 5q spinal muscular atrophy

Executive function is inversely correlated with physical function: the cognitive profile of adult Spinal Muscular Atrophy (SMA)

2021 . Mix, Lucas | Schreiber-Katz, Olivia | Wurster, Claudia D. | Uzelac, Zeljko | Platen, Sophia | Gipperich, Christina | Ranxha, Gresa | Wieselmann, Gary | Osmanovic, Alma | Ludolph, Albert C. | Petri, Susanne | Lulé, Dorothée

Molekulare Therapien bei neuromuskulären Erkrankungen im Kindesalter – Große Hoffnungen und unbekannte Risiken

2020 . Blaschek, Astrid | Vill, Katharina | Müller-Felber, Wolfgang | Schara, Ulrike