Article

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

2022-10-24

Abstract excerpt

<title>Abstract</title> <p>Background Management and treatment of spinal muscular atrophy (SMA) has changed in recent years due to the introduction of novel transformative and potentially curative therapies resulting in the emergence of new disease phenotypes. Yet, little is known about the uptake and impact of these therapies in real-world clinical practice. The objective of this study was to describe current m...

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Literature Corpus work: fcf87180-bf9a-543e-9326-7dc1eb8bc300
DOI: 10.21203/rs.3.rs-2142996/v1

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