Challenges and opportunities in the field of spinal muscular atrophy, including how new disease-modifying therapies are likely to change SMA’s course over time.
Developing an Alzheimer’s disease drug has been notoriously expensive and high risk. Part of the problem has been the difficulty in identifying the right patients for clinical trials and at the right time – that is, those patients showing early signs of cognitive impairment who will progress to AD. However, new diagnostics, biomarkers, and digital...