Commentary on the 2021 update of the KDIGO clinical practice guideline for management of blood pressure in chronic kidney disease.

The 2021 KDIGO clinical practice guideline for the management of blood pressure (BP) in chronic kidney disease (CKD) provided significant practice-changing recommendations for the care of both adult and paediatric CKD patients not receiving dialysis. The purpose of this review is to contextualise these recommendations and evaluate their applicability to the Australian and New Zealand context. Key updates presented in this guideline relate to measurement techniques, with a strong recommendation for standardised office BP measurement, as opposed to routine office BP measurement.

A Phase 1B Trial in GBA1-Associated Parkinson's Disease of BIA-28-6156, a Glucocerebrosidase Activator.

Background: Loss-of-function mutations in the GBA1 gene are one of the most common genetic risk factors for onset of Parkinson's disease and subsequent progression (GBA-PD). GBA1 encodes the lysosomal enzyme glucocerebrosidase (GCase), a promising target for a possible first disease-modifying therapy. LTI-291 is an allosteric activator of GCase, which increases the activity of normal and mutant forms of GCase.

A Biomarker Study in Patients with GBA1-Parkinson's Disease and Healthy Controls.

Background: Molecules related to glucocerebrosidase (GCase) are potential biomarkers for development of compounds targeting GBA1-associated Parkinson's disease (GBA-PD).

Objectives: Assessing variability of various glycosphingolipids (GSLs) in plasma, peripheral blood mononuclear cells (PBMCs), and cerebrospinal fluid (CSF) across GBA-PD, idiopathic PD (iPD), and healthy volunteers (HVs).

Methods: Data from five studies were combined.

Caring for Australians and New Zealanders With Kidney Impairment Guidelines: Rapid Development of Urate Lowering Therapy Guidelines for People With CKD.

Introduction: The slow transformation of new research findings into clinical guidelines is a barrier to providing evidence-based care. The Caring for Australians and New Zealanders with Kidney Impairment (CARI) guidelines are developing models to improve guideline production, one methodology involves more functional concordance between trial groups, such as the Australian Kidney Trials Network (AKTN) and CARI. The objective of this project was to rapidly produce an evidence-based guideline on urate-lowering therapy in patients with chronic kidney disease (CKD), in response to new clinical trial publications on the topic by the AKTN.

A randomized single and multiple ascending dose study in healthy volunteers of LTI-291, a centrally penetrant glucocerebrosidase activator.

Aims: A mutation in the GBA1 gene is the most common genetic risk factor for developing Parkinson's disease. GBA1 encodes the lysosomal enzyme glucosylceramidase beta (glucocerebrosidase, GCase) and mutations decrease enzyme activity. LTI-291 is an allosteric modulator of GCase, enhancing its activity.