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Oct 2026

In vivo gene editing with YOLT-201 for transthyretin amyloid cardiomyopathy – an open-label, first-in-human clinical trial

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive and life-threatening disease driven by continuous hepatic production of misfolded transthyretin. Existing therapies slow progression but require chronic administration. YOLT-201 is an in vivo CRISPR-Cas9 gene-editing therapy delivered by lipid nanopa...

X. Guo, H. Yan, J.-Y. Liao et al. · 0 citations
Open access Oct 2026

Rapid Serum IgG Reduction as a Potential Indicator of Short-Term Clinical Improvement in AChR-Ab-Positive Generalized Myasthenia Gravis: A Post Hoc Analysis of a Phase III Batoclimab Trial.

INTRODUCTION Generalized myasthenia gravis (gMG) is an autoimmune disorder often associated with anti-acetylcholine receptor antibodies (AChR-Abs). Identifying short-term predictors or indicators of clinical improvement is crucial for optimizing treatment strategies. METHODS This post hoc analysis utilized data from...

Zhi-Jun Li, Q. Ke, Jian-Ying Xi et al. · 0 citations

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