Patients’ Perceptions of Nusinersen Effects According to Their Responder Status

Patients’ Perceptions of Nusinersen Effects According to Their Responder Status

Background and Objective: Patients with spinal muscular atrophy (SMA) treated with a disease-modifying therapy (DMT) are often classified as responders or non-responders based on the attainment of a specific improvement threshold on validated functional scales. This categorization may significantly...

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Bibliographic Details
Main Authors:	Lilien, C, Vrscaj, E, Thapaliya, G, Deconinck, N, De Waele, L, Duong, T, Haberlová, J, Kumhera, M, Peirens, G, Szabo, L, Tahon, V, Tang, WJ, Benmhammed, N, Médard, L, Servais, L
Format:	Journal article
Language:	English
Published:	MDPI 2024

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