Rare diseases
Diseases affecting a small percentage of the population.
Overview
Diseases affecting a small percentage of the population.
Key developments include new york confirms first case of rare tick-borne bourbon virus in long island man, silence therapeutics reports positive phase 2 trial results for divesiran in rare blood cancer, z-basivarsen shows sustained benefits in myotonic dystrophy type 1 trial, among other significant events and policy changes.