Areas of improvement in the medical care of SMA: evidence from a nationwide patient registry in Germany

Leibrock, B; Landfeldt, E; Hussong, J; Huelle, T; Mattheus, H; Thiele, S; Walter, MC; Zemlin, M; Moehler, E; Dillman, U; Abner, S; Flotats-Bastardas, M

Flotats-Bastardas, M (通讯作者)，Saarland Univ Hosp, Dept Neuropaediat, Homburg, Germany.

ORPHANET JOURNAL OF RARE DISEASES, 2023; 18 (1):

Abstract

Background Management and treatment of spinal muscular atrophy (SMA) has changed in recent years due to the introduction of novel transformative and p......

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