fig2

First, do no harm: the role of preclinical animal models in predicting adverse events in gene therapy clinical trials for Duchenne muscular dystrophy and X-Linked myotubular myopathy

Figure 2. Diagram depicting DMD gene mutations associated with SAEs. Panels A, B, and C show the dystrophin structure, microdystrophin exon content of therapeutic transgenes, and patient mutations, respectively. From Bönnemann et al., 2023[31]. Republished under the Rights Link for Scientific Communications through the Copyright Clearance Center. SAEs: Severe adverse events; DMD: Duchenne muscular dystrophy.

Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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