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Parent/caregiver perspectives of meaningful improvement in functional domains for people with CDKL5 deficiency disorder: a mixed-methods study

CDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy. Greater understanding of the smallest meaningful improvements f…

Translating multi-omics into healthcare: requisites for scalable and equitable implementation

Multi-omics in combination with advanced computational methodologies synthesizes diverse omics data to provide deeper insights into molecular interac…

Insights from a trial of narrowband UVB for early multiple sclerosis

There is a greater prevalence of multiple sclerosis (MS), a neurological autoimmune condition, in populations living further from the equator, hypoth…

Perth researchers put rare diseases at the centre of global health innovation

Recently, researchers from The Kids Research Institute Australia, Perth Children’s Hospital and The University of Western Australia contributed to th…

Maternal and neonatal outcomes after infection with monkeypox virus clade I during pregnancy in DR Congo: a pooled, prospective cohort study

Monkeypox virus (MPXV) has been linked to vertical transmission, but systematic data are scarce. We aimed to describe the sociodemographic, clinical,…

Australia’s top 10 rare disease research priorities: a priority setting partnership

The aim of this study was to identify and prioritise the ten most important unanswered themes in rare disease research in Australia by integrating pe…

What is it like living with X-linked hypophosphatemia?: results from an Australian consumer survey

X-linked hypophosphatemia (XLH) is a rare, X-linked dominant condition with a high burden of both physical and psychosocial disease. This study aimed…

Major donation to change the landscape of rare and undiagnosed disease

The Kids Research Institute Australia is proud to be a part of the Rare Care Comprehensive Centre (RCCC) for children with rare and undiagnosed disea…

The diagnostic odyssey for children living with a rare disease – Caregiver and patient perspectives: A narrative review with recommendations

Children living with a rare disease often endure a lengthy journey to diagnosis, commonly referred to as a diagnostic odyssey. This journey significa…