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FDA Patient-Focused Drug Development Guidances: Considerations for Trial Readiness in Rare Developmental and Epileptic Encephalopathies

Developmental and epileptic encephalopathies (DEE) are rare, often monogenic neurodevelopmental conditions. Most affected individuals have refractory seizures. All have multiple severe impairments which can be as life-limiting as or more limiting than the seizures themselves. Mechanism- and gene-targeted therapies for these individually rare, genetic conditions hold hope for treatment, amelioration of disease expression, and even cure. 

A Comparison of Inertial Measurement Units and Overnight Videography to Assess Sleep Biomechanics

The assessment of sleep biomechanics (comprising movement and position during sleep) is of interest in a wide variety of clinical and research settings. However, there is no standard method by which sleep biomechanics are measured. This study aimed to (1) compare the intra- and inter-rater reliability of the current clinical standard, manually coded overnight videography, and (2) compare sleep position recorded using overnight videography to sleep position recorded using the XSENS DOT wearable sensor platform.

Evaluation Tools Developed for Rett Syndrome

Rett syndrome (RTT) is a complex neurodevelopmental X-linked disorder associated with severe functional impairments and multiple comorbidities. There is wide variation in the clinical presentation, and because of its unique characteristics, several evaluation tools of clinical severity, behavior, and functional motor abilities have been proposed specifically for it.

Strengths and challenging behaviors in children and adolescents with Prader-Willi syndrome: Two sides to the coin

Prader-Willi Syndrome (PWS) is a rare genetic disorder associated with emotional/behavioral disturbances. These difficulties are well documented in the literature, but the positive attributes of these individuals are not described.

Gina Arena

Program Manager, PLAYCE team

Nasal spray to prevent ear infections closer to fruition thanks to major grant

A nasal spray that could potentially prevent childhood ear infections and reduce antibiotic use is a step closer to clinical trials thanks to a $500,000 CUREator grant.

Linkage of the Australian Childhood Immunisation Register (ACIR) and state-based registers to evaluate and inform Australia’s immunisation program

Christopher Hannah Tom Blyth Moore Snelling MBBS (Hons) DCH FRACP FRCPA PhD OAM BSc (Hons) GradDipClinEpi PhD BMBS DTMH GDipClinEpid PhD FRACP Centre

Nature Play & Grow

Helping families to unlock the mental and physical health benefits of connecting with nature and community through outdoor play - easily, and locally.

Investigation of trimethoprim/sulfamethoxazole resistance in an emerging sequence type 5 methicillin-resistant Staphylococcus aureus clone reveals discrepant resistance reporting

Our findings are crucial in demonstrating that the Northern Territory STS clone is not STX resistant

Whole genome sequencing reveals extensive community-level transmission of group A Streptococcus in remote communities

There was evidence of extensive transmission of group A Streptococcus both within households and across the community