Comparative Effectiveness of Valoctocogene Roxaparvovec and Prophylactic Factor VIII Replacement in Severe Hemophilia A.

Introduction: A prospective, non-interventional study (270-902) followed 294 adults with severe hemophilia A (SHA) receiving prophylactic factor VIII (FVIII). From these participants, 112 rolled over into a single-arm, multicenter, phase 3 trial (GENEr8-1; NCT03370913) that evaluated efficacy and safety of valoctocogene roxaparvovec, a gene therapy that provides endogenous FVIII in individuals with SHA. Participants from 270-902 who did not roll over provide an opportunity for a contemporaneous external control.

Spatiotemporal dynamics of Phormidium cover and anatoxin concentrations in eight New Zealand rivers with contrasting nutrient and flow regimes.

Toxic benthic cyanobacterial proliferations, particularly of the genus Phormidium, are a major concern in many countries due to their increasing extent and severity. The aim of this study was to improve the current understanding of the dominant physicochemical variables associated with high Phormidium cover and toxin concentrations. Phormidium cover and anatoxin concentrations were assessed weekly for 30weeks in eight predominately cobble-bed rivers in the South Island of New Zealand.

The rise of toxic benthic Phormidium proliferations: A review of their taxonomy, distribution, toxin content and factors regulating prevalence and increased severity.

There has been a marked increase in the distribution, intensity and frequency of proliferations of some species of the benthic mat-forming, toxin-producing genus Phormidium in rivers globally over the last decade. This review summarises current knowledge on their taxonomy, distribution, toxin content, environmental drivers of proliferations, and monitoring and management strategies in New Zealand. Although toxic Phormidium proliferation occurs in rivers worldwide little is known about these factors in most countries.