Patient Preferences in Rare Diseases: A Qualitative Study in Neuromuscular Disorders to Inform a Quantitative Preference Study.

Introduction: It has become increasingly important to include patient preference information in decision-making processes for drug development. As neuromuscular disorders represent multisystem, debilitating, and progressive rare diseases with few treatment options, this study aimed to explore unmet health care needs and patient treatment preferences for two neuromuscular disorders, myotonic dystrophy type 1 (DM1) and mitochondrial myopathies (MM) to inform early stages of drug development.

Methods: Fifteen semi-structured interviews and five focus group discussions (FGDs) were held with DM1 and MM adult patients and caregivers.

Leader psychobiography and social movement studies: a Kleinian case study of Bruce Gagnon and the outer space protection movement.

This paper begins by highlighting the necessity of combining sociological and psychoanalytic approaches in the study of social movements, but acknowledges that psychobiographical studies of prominent movement leaders sit uneasily within the sociological tradition. The author attempts to illustrate that leader psychobiography can make a contribution to understanding social movements, however, provided it offers a way into understanding broader psychosocial issues within the movement. This is achieved through a psychobiographical portrait of Bruce Gagnon, a leader within the outer space protection movement.