Bone Abstracts

Objectives: Achondroplasia (ACH), the most common form of human dwarfism, is caused by a gain-of-function mutation in the fibroblast growth factor receptor 3 (FGFR3) gene, a key negative regulator of endochondral ossification. C-type natriuretic peptide (CNP) inhibits the FGFR3 pathway and thereby promotes proliferation and differentiation of chondrocytes to promote bone growth. TransCon CNP is a prodrug designed to provide continuous exposure to CNP to optimize efficacy with ...

Objectives: Children with Duchenne muscular dystrophy (DMD) receiving long-term glucocorticoid (GC) therapy are at risk for osteoporosis and fragility fractures. Recent studies showed that cortical thickness and areas were associated with increased fracture risk. Digital X-ray measurement of the cortical thickness of the metacarpal bones has a potential role as a marker for bone health in children, but has not been evaluated in DMD. The aim of this study was to compare bone ag...

A randomized, placebo-controlled phase 2 study evaluated the safety and efficacy of BA058, an analog of hPTHrP(1–34), in postmenopausal women with severe osteoporosis. 221 patients were randomized to received BA058 20, 40, and 80 μg, placebo or teriparatide (TP) 20 μg, by daily s.c. injection. 184 (83%) patients completed an initial 24 weeks of treatment. The mean percent change in lumbar spine BMD at 24 weeks was 1.6% with placebo, 6.7% with BA058 80 μg, a...

Tissue healing/reconstruction as well as exacerbated inflammatory diseases may benefit from stem cell based therapies. Ex vivo isolated tissue mesenchymal stromal cells (MSCs) displaying multi-potent activity and immune-regulatory functions were long ago proposed as therapeutic cells and already tested in many clinical assays. Nevertheless, their use may be restricted because of the few number that can be recovered from adult tissues, their limited in vitro e...

Tissue healing/reconstruction as well as exacerbated inflammatory diseases may benefit from stem cell based therapies. Ex vivo isolated tissue mesenchymal stromal cells (MSCs) displaying multi-potent activity and immune-regulatory functions were long ago proposed as therapeutic cells and already tested in many clinical assays. Nevertheless, their use may be restricted because of the few number that can be recovered from adult tissues, their limited in vitro e...

Objective: Bone and muscle deficits are observed in patients with Crohn’s disease (CD). High-impact exercise (HIE), such as jumping based exercise, can provide hypertrophic and osteogenic stimulus, however to date there have been no studies of HIE in CD. This study aimed to assess the acceptability and feasibility of participating in HIE in adolescents and adults with CD.Methods: Two anonymous questionnaires surveyed adolescents and adults, respecti...

Objectives: Bone development can be enhanced by exercise during growth, but not all types of exercise may be beneficial. Weight bearing and non-weight bearing sports have different effects on bone outcomes during adolescence. However, there is no longitudinal evidence comparing the effects of popular sports in the UK on bone acquisition in adolescent males.Methods: In the present study 116 adolescent males (13.1±0.1 years: 37 footballers, 37 swimmer...

BA058 is being developed as an anabolic therapy for the treatment of osteoporosis. Daily BA058 SC injection has produced promising safety and efficacy results in early clinical studies, and is currently enrolling in a Phase 3 fracture prevention study. There is, however, a significant need for an alternative to injection that improves patient convenience and compliance. We have investigated the use of a solid Microstructured Transdermal System (3M) for transdermal (TD) deliver...

Objective: This multi-objective prospective observational study is intended to examine treatment patterns, occurrence of gastrointestinal (GI) complications and clinical and health outcomes in post-menopausal women with osteoporosis (OP) in Europe and Canada. This abstract presents the baseline results of the treated population.Materials and methods: Post-menopausal osteoporotic women currently on treatment completed surveys intended to measure the incid...

Objective: This multi-objective prospective study (MUSIC-OS) is intended to examine treatment patterns, occurrence of gastrointestinal (GI) complications and clinical and health outcomes in post-menopausal osteoporotic (OP) women in Europe and Canada. Surveys were administered to both physicians and patients as part of the MUSIC-OS study to document reasons for undertreatment in OP from both physician and patient perspectives.Materials and methods: A phy...