Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for β-thalassemia.

autologous hematopoietic stem cell lentiviral-based gene therapy with betibeglogene autotemcel has been studied in patients with transfusion-dependent β-thalassemia in Phase III clinical trials (HGB-207 and HGB-212), with 90% of patients reaching transfusion independence (TI). Here, we explore manufacturing parameters, drug product quality attributes, and limited patient characteristics that had an impact on clinical efficacy in HGB-207 and HGB-212. Retrospective analysis revealed that the peripheral blood vector copy number (VCN) was related to TI, with a strong correlation between peripheral blood VCN at 6 months and gene therapy-derived therapeutic protein (HbA) expression at 6 months (correlation coefficient, 0.

Total Keratometry Measured With a Swept-Source Optical Biometer Versus Anterior Keratometry: From Planning to Postoperative Results.

Purpose: To compare the influence of total keratometry (TK) versus anterior keratometry (K) measured with the swept-source optical biometer IOLMaster 700 (Carl Zeiss Meditec AG) on the planning of toric intraocular lenses (IOLs), and the error in predicted residual astigmatism (PRA).

Methods: This single-center retrospective study included 247 eyes of 180 patients. In eyes undergoing cataract surgery, the optimal toric IOL was calculated based on K or TK measured with the IOLMaster 700.

Identifying best-practice features of diabetic retinopathy treatment models for Aboriginal and Torres Strait Islander Australians.

Objective: Indigenous Australians are nearly three times more likely to have diabetes than non-Indigenous Australians. The prevalence of diabetes-related vision impairment for Indigenous Australians is 5.5% compared to 1.

ACVR and ECVDI consensus statement for the standardization of the abdominal ultrasound examination.

This consensus statement is designed to provide a standard of care document and describes the ACVR and ECVDI definition for performing a standard abdominal ultrasound examination in dogs and cats. The ACVR and ECVDI define a standard abdominal ultrasonographic examination as a complete exam of the abdominal organs which is appropriately documented. The consensus statement intends to provide guidance to veterinary sonographers and veterinarians for the performance and documentation of high-quality diagnostic ultrasound examination.

Multimodal Imaging of Unilateral Acute Maculopathy Associated with Hand, Foot, and Mouth Disease: A Case Series.

We described clinical and multimodal imaging findings in 4 patients with unilateral acute idiopathic maculopathy (UAIM) associated with hand, foot, and mouth disease. Four eyes of 4 patients (3 women and 1 man) with a mean age of 35 years (range: 24-40 years) were included. A bacillary detachment was observed in 3 out of the 4 eyes and was strongly suspected in the remaining eye.

MACULAR ATROPHY AFTER MACULAR HOLE SURGERY ASSISTED BY MEMBRANE BLUE DUAL: A CASE SERIES.

Purpose: The aim of this study is to describe the clinical and multimodal imaging findings in patients with macular atrophy after macular hole surgery assisted by Membrane Blue Dual.

Method: This study is a monocenter, retrospective, observational case series that included patients who presented with macular atrophy following macular hole surgery.

Results: Among the patients included in this study, four were operated for idiopathic macular hole and one for total retinal detachment associated with macular hole.

Lessons learned from a subsidised spectacles scheme aiming to improve eye health in Aboriginal people in Victoria, Australia.

The Victorian Aboriginal Spectacles Subsidy Scheme (VASSS) aimed to improve access to visual aids and eye care for Aboriginal and Torres Strait Islander Victorians. The VASSS started in July 2010 and has operated continually since. In 2016, we explored the collaborations, planning, adaptations and performance of the VASSS over the first 6 years by reviewing and analysing service data, as well as data from semistructured interviews, focus groups and surveys.

Cataract surgery and Indigenous eye care: A review.

Cataract remains the leading cause of blindness in Aboriginal and Torres Strait Islander peoples and is still a major cause of vision loss. The pathway of care to cataract surgery has many potential gaps and barriers. Although there has been a significant increase in services over the last few years, there is still the urgent need to facilitate timely and affordable cataract surgery.

Diagnostic imaging for the assessment of acquired abdominal vascular diseases in small animals: A pictorial review.

Advances in interventional radiology and surgical techniques now allow complex abdominal diseases to be more successfully treated in small animals. Abdominal vascular alterations, acquired as individual process or as complication of other lesions such as neoplasia, can be life-threatening or at least greatly limit curative interventions of underlying diseases. Computed tomography (CT) and high-definition ultrasonography are now readily available in veterinary referral centers.

Management of drooling in children with cerebral palsy: A French survey.

Aim: To characterise children with cerebral palsy (CP) and pathological drooling in France, and to describe care pathways, assessment and treatment.

Method: A transversal, observational, descriptive survey of the practices and opinions of 400 health professionals potentially involved in the care of children with CP, was carried out nationally across France in 2013.

Results: The response rate was 36%.

Ultrasonographic and clinicopathological features of pyloroduodenal adenomatous polyps in cats.

Objectives The aim of the study was to report the ultrasonographic and clinicopathological findings in cats with confirmed pyloroduodenal adenomatous polyps. Methods Clinicopathological data, ultrasonographic and histopathological findings were collected retrospectively from medical records. Results Pyloroduodenal polyps appeared as small moderately echogenic and homogeneous nodules filling most of the proximal duodenal or pyloroduodenal lumen.

Breaking down barriers to eye care for Indigenous people: a new scheme for delivery of eye care in Victoria.

This report describes the implementation of and outcomes from a new spectacle subsidy scheme and de-centralised care options for Aboriginal and Torres Strait Islander peoples in Victoria, Australia. The Victorian Aboriginal Spectacle Subsidy Scheme (VASSS) commenced in 2010, as an additional subsidy to the long-established Victorian Eyecare Service (VES). The Victorian Aboriginal Spectacle Subsidy Scheme aimed to improve access to and uptake of affordable spectacles and eye examinations by Indigenous Victorians.