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Good morning! Today, we discuss the woman who spearheaded progress in multiple myeloma for decades, see mixed but promising results from Sanofi’s multiple sclerosis drug, and hear why All of Us should not be defunded.
The need-to-know this morning
- Dyne Therapeutics reported updated results from an early stage study of its experimental treatment for Duchenne muscular dystrophy amenable to exon 51 skipping. The company also announced the departures of its chief medical officer, chief operating officer, and chief business officer.
- Vaxcyte reported results from a clinical trial of its experimental pneumococcal vaccine in adults.
- Denali Therapeutics said it reached an agreement with the FDA for an accelerated approval filing in early 2025 of its treatment for Hunter syndrome.
How an exec-turned-patient helped revolutionize multiple myeloma treatment
In 1996, Kathy Giusti was diagnosed with multiple myeloma and given just three years to live. One reason she’s still alive today: The former drug company executive founded the Multiple Myeloma Research Foundation in 1998, which has since raised more than $600 million for research, launched nearly 100 clinical trials, and helped bring more than 15 new drugs to market, STAT’s Matthew Herper writes. Though her illness and work took a heavy toll on her family, the progress is stunning, given that multiple myeloma is a relatively rare cancer.
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