Antisense oligonucleotides are reshaping drug design, from RNase H gapmers to inhaled delivery; this FAQ explains how they work and why trials still fail.
Bristol Myers Squibb's Nathan Pennell explains how the MRD endpoint supports accelerated approval in multiple myeloma without changing how clinicians monitor patients.
New clinical milestones, manufacturing investments, and regulatory incentives could help developers overcome barriers to delivering treatments for patients with rare diseases.