Phase I/II data for its ADC candidates in myotonic dystrophy type 1 and Duchenne muscular dystrophy indicate potential for biomarker-based accelerated approval in both diseases, Dyne asserts.

Public Company Edition: Discussions at the J.P. Morgan Healthcare Conference 8-11 January in San Francisco should offer some insight on industry’s and investors’ expectations about the financial markets in 2024. The year started with some big financings, but also some new job cuts.

Move over big data, a biostatistician calls for more people to think small for rare diseases and FDA officials indicate openness to the trial approaches laid out.

A pivotal miss leaves the company more reliant on earlier-stage pipeline programs as it talks to regulators about the path forward for its lead product.