Endocrine Abstracts

Abaloparatide (ABL), a synthetic PTHrP(1–34) analogue (75% homology with PTHrP), has high affinity for the RG subtype of the PTH1R and low affinity for the R0 conformation, resulting in a greater stimulus to bone formation vs resorption. In animals, ABL increases bone formation markers with minimal resorption marker increase, increases bone mass, improves microarchitecture and bone strength. In the Phase 3 Abaloparatide Comparator Trial in Vertebral Endpoints s...

We are living through an exciting period during which new medical treatments are emerging for a range of rare bone diseases. These include monoclonal antibodies, small molecules and repurposed as well as truly innovative drugs. Whilst underlying the use of them all is an increased understanding of some aspect of bone biology, presently each sits at a different point on the pathway from bench to bedside. Asfotase alfa (bone-targeted alkaline phosphatase) is licensed for the tre...

Genetic discoveries in patients and families with rare bone disorders have highlighted the complexity of molecular mechanisms and cellular pathways governing normal bone development and homeostasis. More than 400 different forms of skeletal dysplasia have been described. Most of them result from single-gene defects, involving genes that are of major importance for a particular cellular event in skeletal development. Studies aiming to identify the involved pathways can enable d...