A5078684328- Connective tissue disorders research
- Lipid metabolism and disorders
- Neonatal Respiratory Health Research
- Congenital Diaphragmatic Hernia Studies
- Hip disorders and treatments
- Pancreatic and Hepatic Oncology Research
- Aldose Reductase and Taurine
- Pharmaceutical studies and practices
- Hip and Femur Fractures
- Pelvic and Acetabular Injuries
- Pancreatitis Pathology and Treatment
- Orthopaedic implants and arthroplasty
- Bone fractures and treatments
- Aortic aneurysm repair treatments
- Pediatric Hepatobiliary Diseases and Treatments
- Fibroblast Growth Factor Research
- Retinopathy of Prematurity Studies
- Orthopedic Infections and Treatments
- Hernia repair and management
- Spinal Fractures and Fixation Techniques
- Pregnancy-related medical research
- Neonatal Health and Biochemistry
- Blood Pressure and Hypertension Studies
- Infant Nutrition and Health
- Neuroendocrine Tumor Research Advances
Instituto de Investigación Biomédica de Málaga
2022-2025
Hospital Clínico Universitario Virgen de la Victoria
2016-2025
Andalusian Centre for Nanomedicine and Biotechnology
2022-2024
Murdoch Children's Research Institute
2021-2024
Hospital Research Foundation
2024
Royal Children's Hospital
2021
The University of Melbourne
2021
Achondroplasia is caused by pathogenic variants in the fibroblast growth factor receptor 3 gene that lead to impaired endochondral ossification. Vosoritide, an analog of C-type natriuretic peptide, stimulates bone and development for treatment achondroplasia. This phase extension study was conducted document efficacy safety continuous, daily vosoritide children with achondroplasia, two-year results are reported.
Achondroplasia is the most common short-limbed skeletal dysplasia resulting from gain-of-function pathogenic variants in fibroblast growth factor receptor 3 (FGFR3) gene, a negative regulator of endochondral bone formation. Most treatment options are symptomatic, targeting medical complications. Infigratinib an orally bioavailable, FGFR1-3 selective tyrosine kinase inhibitor being investigated as direct therapeutic strategy to counteract FGFR3 overactivity achondroplasia.The main objective...
Achondroplasia, caused by a pathogenic variant in the fibroblast growth factor receptor 3 gene, is most common skeletal dysplasia. The Lifetime Impact of Achondroplasia Study Europe (LIAISE; NCT03449368) aimed to quantify burden achondroplasia among individuals across broad range ages, including adults.Demographic, clinical and healthcare resource use data were collected from medical records patients enrolled 13 sites six European countries this retrospective, observational study....
Background: Guided bone regeneration (GBR) is a widely used technique in oral and maxillofacial surgery to restore lost bone. The aim of this study evaluate the effectiveness titanium occlusive barriers GBR for increasing volume both vertical horizontal dimensions. Methods: A prospective analysis was conducted on 11 patients (15 cases) undergoing augmentation with combined graft biomaterials dental implant placement. Bone gain assessed using pre- postoperative low-dose cone beam computed...
Achondroplasia is a genetic skeletal condition that results in disproportionately short stature and medical complications throughout life. Infigratinib an orally bioavailable FGFR1-3 selective tyrosine kinase inhibitor development for achondroplasia.
Vosoritide, a potent stimulator of endochondral bone growth, is approved for use in children with achondroplasia (ACH) until closure epiphyses. A 52-week, phase 3, pivotal trial demonstrated significant improvement annualized growth velocity (AGV) vosoritide compared to placebo ACH aged 5-18 years. This an analysis available long-term data the ongoing 3 open-label extension study.
Vosoritide, a potent stimulator of endochondral bone growth, is approved for use in children with achondroplasia (ACH) until closure epiphyses. A 52-week, phase 3, pivotal trial (111-301) demonstrated significant improvement annualized growth velocity (AGV) vosoritide compared to placebo ACH aged 5-18 years. We report data from Quality Life Short Stature Youth (QoLISSY) questionnaires collected treated study 111-301 and its ongoing open-label extension 111-302.
Abstract Disclosure: R. Savarirayan: Advisory Board Member; Self; Ascendis Pharma, BioMarin, QED Therapeutics, Sanofi. Consulting Fee; BioMarin. Grant Recipient; Theracon. Research Investigator; J. De Bergua: None. P. Arundel: Salles: V. Saraff: B. Delgado: A. Leiva-Gea: H. McDevitt: M.P. Nicolino: M. Rossi: Salcedo: Cormier-Daire: Skae: Kannu: Novartis, Ipsen. CIHR. M.B. Bober: Biomarin. Pfizer, Therapeutics. Phillips: Saal: Alexion. Alexion, Harmatz: Audentes, Aeglea, Homology, JCR,...
Although dual implant constructs have recently been explored with promising results in very distal periprosthetic femur fractures (PPKF), the gold standard treatment of Rorabeck and Taylor type III PPKF remains a replacement or highly constrained rotating hinge implant. However, this surgery is aggressive expensive for functionally low-demanding elderly patients. A new surgical technique using locking plates polymethyl methacrylate cement augmentation described to retain femoral component...
Vosoritide is a C-type natriuretic peptide analog that addresses an underlying pathway causing reduced bone growth in achondroplasia. Understanding the vosoritide treatment effect requires evaluation over extended duration and comparison with outcomes untreated children.
La acondroplasia requiere un seguimiento multidisciplinario, con el objetivo de prevenir y manejar las complicaciones, mejorar la calidad vida favorecer su independencia e inclusión social. Esta revisión se justifica por múltiples publicaciones generadas en los últimos años que han llevado a cabo cambio gestión. Se desarrollado diferentes guías recomendaciones, entre destacan realizada Academia Americana Pediatría 2005 recientemente actualizada (2020), guía japonesa primer Consenso Europeo...
Abstract Objectives: Vosoritide is a potent stimulator of endochondral bone growth and in development for the treatment achondroplasia, most common form disproportionate short stature. We previously reported on 52-week, phase 3, pivotal study that demonstrated highly statistically significant improvement annualized velocity (AGV) when vosoritide was compared to placebo children with achondroplasia aged 5-18 years (Savarirayan et al, Lancet, 2020). This an analysis data after additional 52...