To identify changes in the proteome associated with onset and progression of ATTRv amyloidosis, we performed an observational, case-controlled study which compared proteomes of patients with ATTRv amyloidosis and healthy controls. Plasma levels of >1,000 proteins were measured in patients with ATTRv amyloidosis with polyneuropathy who received either placebo or patisiran in the APOLLO study and in healthy controls. The impact of patisiran on the time profile of each protein was determined by linear mixed...

Objective To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Methods Part 1 was a randomized, double-blind, placebo-controlled, 12-week dose titration of once-weekly golodirsen; Part 2 is an ongoing, open-label evaluation. Safety and pharmacokinetics were primary and secondary objectives of Part 1. Primary biological outcome measures of part 2 were blinded exon skipping and dystrophin protein production on muscle biopsies (baseline,...

The avian lung is highly specialized and is both functionally and morphologically distinct from that of their closest extant relatives, the crocodilians. It is highly partitioned, with a unidirectionally ventilated and immobilized gas-exchanging lung, and fully decoupled, compliant, poorly vascularized ventilatory air-sacs. To understand the evolutionary history of the archosaurian (birds, crocodilians and their common ancestors) respiratory system, it is essential to determine which anatomical characteristics are shared between birds and crocodilians and the role...

This study investigated individuals’ preference structures for workplace coaching providers. Guided by questions about relative weightings of seven important coach(ing) characteristics (i.e., coach work experience/background/gender; coaching training; personal recommendations; client feedback; coaching cost), we carried out a conjoint analysis, using a mixed occupational sample (N = 383). In addition, we conducted linear regression analyses to determine the extent to which coaches’ perceived competence, likeability and trustworthiness might impact on individuals’ decision-making processes. Potential coachees favoured...