Bone Abstracts

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...

Introduction: Osteogenesis imperfecta (OI) is characterized by low bone mass, skeletal fragility and, frequently, short stature. We previously showed in oim/oim mice that sclerostin inhibition increased bone mass, mineral content and strength. Here, we compared the body length and the sizes of long bones, head and vertebrae between oim/oim and wildtype mice and analyzed the effect of sclerostin antibody (Scl-Ab) on these parameters.Materials and methods:...

Proteoglycans are abundant and structurally complex bio macromolecules. They reside on the cell surface and are a major component of the extracellular matrix including bone. Defective formation of proteoglycans may case pleiotropic connective tissue syndromes including EDS-like and Larsen syndrome–like phenotypes.We report on a girl with disproportionate short stature and joint laxity with pes planus and radial head dislocation. She was previously a...

Background: Congenital hypoparathyroidism in children is a condition with diverse genetic etiologies. Kenny-Caffey syndrome (KCS) is a rare genetic disorder characterized by hypoparathyroidism, short stature, cortical thickening and medullary stenosis of the tubular bone and dysmorphic features including micrognathia, prominent forehead and eye abnormalities. The autosomal dominant form of KCS [KCS type 2(KCS2)] is differenced from autosomal recessive form of KCS [KCS type 1 (...

ACH (achondroplasia) is one of the most common skeletal dysplasias with severe short stature caused by gain-of-function mutations in the FGFR3 gene. Foramen magnum stenosis is a serious neurological complication of ACH. Downregulation of the FGFR3 signaling is a radical therapeutic strategy for the disease. We previously demonstrated that meclozine, an over-the-counter drug for motion sickness, inhibited elevated FGFR3 signaling in chondrocytic cell lines. In the present study...

Objectives: We have performed a novel cell therapy using culture-expanded bone marrow cells (BMC) and platelet rich plasma (PRP) during limb lengthening procedure since 2002. In the present study, we evaluated the efficacy of the cell therapy on new bone regenerates in patients with achondroplasia (ACH) and hypochondroplasia (HCH).Methods: The transplantation technique of BMC and PRP was described previously (Bone 40: 522–528, 2007). Inclusion crite...

Objectives: Bisphosphonates are frequently used in children with skeletal disorders, however optimal dosing and regimens are unknown. Early treatment focused on pamidronate (PAM), a second-generation formulation, however use of zoledronate (ZOL), a more potent third-generation bisphosphonate, has recently increased due to shorter and less frequent infusions. The objective of this study is to compare short-term safety of ZOL and PAM in a pediatric population.<p class="abste...

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Tibial dysplasia, which leads to fracture and pseudarthrosis, occurs in around 4% of children with NF1, and also in children with no underlying disorder. Pseudarthrosis of the fibular may or may not be present, or as an isolated entity, as can pseudarthrosis in the forearm (rare). Other bone problems faced by individuals with NF1 are scoliosis (20%), pectus excavatum/carinatum (12%), and sphenoid wing dysplasia (7%). Dural ectasia and plexiform neurofibromas can also affect th...

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