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- AstraZeneca and Ionis see pivotal trial miss for ATTR-CM drug WainuaAstraZeneca announced that its experimental therapy Wainua, developed with Ionis, failed to meet primary endpoints in a pivotal study for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). T…
- Father spends $70 million on gene therapy trial for daughter with ultra-rare disease, faces FDA hurdlesMatt Wilsey has spent a decade and about $70 million trying to develop a gene therapy for his 15-year-old daughter Grace, who suffers from a fatal ultra-rare disease. His personal quest has turned…