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Parental experiences of using continuous glucose monitoring in their young children with early-stage type 1 diabetes: a qualitative interview study

To explore parents' experiences of using continuous glucose monitoring in their young children with early-stage type 1 diabetes, being followed in the Australian Environmental Determinants of Islet Autoimmunity (ENDIA) study.

Coupling of response biomarkers between tumor and peripheral blood in patients undergoing chemoimmunotherapy

Platinum-based chemotherapy in combination with anti-PD-L1 antibodies has shown promising results in mesothelioma. However, the immunological mechanisms underlying its efficacy are not well understood and there are no predictive biomarkers to guide treatment decisions.

Spatial co-distribution of tuberculosis prevalence and low BCG vaccination coverage in Ethiopia

While bacille-calmette-guerin (BCG) vaccination is one of the recommended strategies for preventing tuberculosis, its coverage is low in several countries, including Ethiopia. This study investigated the spatial co-distribution and drivers of TB prevalence and low BCG coverage in Ethiopia.

The Feasibility of Personalized Endpoints in Assessing Treatment Outcomes for Rare Diseases: A Pilot Study of Goal Attainment Scaling in SCN2A-Associated

For individuals living with rare neurodevelopmental disorders, particularly those who are at the most severe end of the spectrum, standardized outcome measures may lack the sensitivity to capture small but meaningful changes. 

Results of an Australian trial of an automated insulin delivery (AID) system and other studies support likely benefit of AID use for many Australian adults with type 1 diabetes

Less than 20% of Australians with type 1 diabetes (T1D) meet recommended glucose targets. Technology use is associated with better glycaemia, with the most advanced being automated insulin delivery (AID) systems, which are now recommended as gold-standard T1D care. Our Australian AID trial shows a wide spectrum of adults with T1D can achieve recommended targets. Other studies, including lived experience data, are supportive. Insulin pumps are not subsidised for most Australian adults with T1D. We advocate change. 

Pushing the boundaries of rare disease diagnostics with the help of the first Undiagnosed Hackathon

Timo Lassmann BSc (Hons) MSc PhD Feilman Fellow; Head, Precision Health Research and Head, Translational Intelligence timo.lassmann@thekids.org.au

Compatibility of pentoxifylline injection with syringe and inline filters

Recent studies have shown that PTX is compatible with a wide range of intravenous medicines used in NICU settings2–4; however, the compatibility of PTX with inline intravenous filters or syringe filters used in aseptic compounding facilities has not previously been reported.

“I just don’t know enough”: Australian perspectives on community involvement in health and medical research

There is increasing global support from governments and other funding bodies for community involvement in research, alongside a scientific and moral imperative for responsible and ethical research practice. Ninety per cent of Australian patient-led organisations in rare diseases have clearly articulated research priorities, indicating a desire among people affected by disease to be involved in research that impacts their communities. 

Using Focussed Ethnography to Observe and Understand the Actions and Interactions of People With Prader-Willi Syndrome When They Exercise at a Community Gym: A Protocol

Exercise for people with Prader-Willi syndrome (PWS) is important for their health and wellbeing and can provide opportunities for community participation. However, they may find it difficult to participate in some contexts, such as community gyms because social and environmental barriers in these settings may compound difficulties caused by physical impairments or intellectual disability.

A global mathematical model of climatic suitability for Plasmodium falciparum malaria

Climatic conditions are a key determinant of malaria transmission intensity, through their impacts on both the parasite and its mosquito vectors. Mathematical models relating climatic conditions to malaria transmission can be used to develop spatial maps of climatic suitability for malaria. These maps underpin efforts to quantify the distribution and burden of malaria in humans, enabling improved monitoring and control.