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The first precision therapy for children with achondroplasia, vosoritide, is now approved in many regions, including Australia, the USA, the European Union and Japan. This article discusses the recent trial results for this therapy regarding growth and the co-morbidities associated with achondroplasia and considers the rationale for its clinical use.
Most women experience vasomotor symptoms during menopause, which considerably affect their quality of life. While vasomotor symptoms can be substantially reduced with menopausal hormone therapy, many women are unable or choose not to take hormone therapy. Now, studies have demonstrated that neurokinin 3 receptor antagonists have a level of effectiveness against vasomotor symptoms that is similar to that of hormone therapy.
Type 2 diabetes mellitus is a progressive disease requiring regular monitoring and therapeutic changes. It is important that healthcare professionals embrace both ends of the spectrum of therapeutic inertia, including appropriate advancement and de-intensification of therapies.
Type 1 diabetes mellitus (T1DM) can be predicted, and immune therapy can alter the progression of the disease. The FDA’s approval of teplizumab as the first disease-modifying therapy for T1DM and the first therapy aimed at delaying the clinical onset of any immune-mediated disease represents a paradigm shift in the treatment of T1DM.
