Consensus statement of the Hungarian Clinical Neurogenic Society about the therapy of adult SMA patients

• Published in: Ideggyógyászati szemle Vol. 74; no. 3-4; pp. 79 - 86
• Main Authors: Boczán, Judit, Klivényi, Péter, Kálmán, Bernadette, Széll, Márta, Karcagi, Veronika, Zádori, Dénes, Molnár, Judit Mária
• Format: Journal Article
• Language: Hungarian
• Published: Hungary 30.03.2021
• Subjects: Adult; Child; Consensus; Humans; Hungary; Muscular Atrophy, Spinal - genetics; Muscular Atrophy, Spinal - therapy; Prospective Studies; Retrospective Studies; Hungary; spinal muscular atrophy; risdiplam; therapy; spinal muscular atrophy (SMA); onasemnogene abeparvovec-xioi

Background - Spinal muscular atrophy (SMA) is an autosomal recessive, progressive neuromuscular disorder resulting in a loss of lower motoneurons. Recently, new disease-modifying treatments (two drugs for splicing modification of SMN2 and one for SMN1 gene replacement) have become available. Purpose...