Too little, too soon: Japan's experiment in regenerative medicine deregulation.

In 2013, Japan established a conditional approval pathway for cell and gene therapies. Our analysis of the four products approved via this pathway identifies evidence suggesting shortcomings in safety and efficacy data submitted by the sponsors and raises concerns about whether this pathway is delivering on its promise.

Approved gene therapies in Australia: coming to a store near you.

Gene therapy has been promising paradigm-shifting advances in medical science for over two decades. Broadly, it is defined as a human therapy in which an existing defective gene function is added to, replaced, edited or disrupted to achieve a clinical benefit, up to and including a potential lifelong cure. Although originally set out to treat monogenic disorders, gene therapy has since been utilised to treat neoplasia, cardiovascular and neurodegenerative diseases, as well as infections.

Malignancy associated hypercalcaemia-responsiveness to IV bisphosphonates and prognosis in a palliative population.

Background: Hypercalcaemia is a potentially fatal paraneoplastic complication of malignancy. It primarily manifests during the advanced phase of cancer, with the life expectancy of patients ranging from weeks to months. The mainstay of treatment is with bisphosphonates, but these are not frequently used in a palliative population due to potential conflict with goals of care.

Heat stroke induced cerebellar dysfunction: A "forgotten syndrome".

We report a case of heat stroke induced acute cerebellar dysfunction, a rare neurological disease characterized by gross cerebellar dysfunction with no acute radiographic changes, in a 61 years old ship captain presenting with slurred speech and gait ataxia. A systematic review of the literature on heat stroke induced cerebellar dysfunction was performed, with a focus on investigations, treatment and outcomes. After review of the literature and detailed patient investigation it was concluded that this patient suffered heat stroke at a temperature less than that quoted in the literature.