Results 11 to 20 of about 534 (161)

Early initiation of therapy with vosoritide: case report. Description of the first Italian patient affected by achondroplasia treated with vosoritide before 2 years of age [PDF]

Frontiers in Pediatrics
Achondroplasia, the most common skeletal dysplasia, is mainly characterized by disproportionate short stature. Affected patients present a reduced linear growth, and the greatest decrease in growth velocity is evident in infancy.
Mila Ann Kalapurackal, Petra Wanker
doaj   +4 more sources

Genetic Bone Diseases: A Scoping Review of Pathology, Symptoms, Diagnosis, Treatment, and New Horizons [PDF]

Advanced Genetics
Genetic bone diseases are a rare group of afflictions suffered by the general population. However, their rarity should not diminish research efforts to help patients understand and treat their diseases.
Colin Jones, Ambalangodage C. Jayasuriya
doaj   +3 more sources

Pharmacokinetics and Exposure–Response of Vosoritide in Children with Achondroplasia [PDF]

Clinical Pharmacokinetics, 2021
BACKGROUND AND OBJECTIVE: Vosoritide, an analog of C-type natriuretic peptide, has been developed for the treatment of children with achondroplasia. The pharmacokinetics of vosoritide and relationships between plasma exposure and efficacy, biomarkers ...
Henshaw, J, Day, J, Larimore, K, Hoover-Fong, J, Qi, Y, Fisheleva, E, Wilcox, WR, Ozono, K, Jeha, G, Savarirayan, R, Cherukuri, A, Mohnike, K, Huntsman-Labed, A, Irving, M, Chan, ML, Jayaram, K, Bacino, CA, Seid, L, Horton, WA, Alice Huntsman-Labed, Lori Seid, Kala Jayaram, Joshua Henshaw, Yulan Qi, Kevin Larimore, Julie Hoover-Fong, Keiichi Ozono, Jonathan Day, Ravi Savarirayan, William A. Horton, Elena Fisheleva, George Jeha, Melita Irving, Ming Liang Chan, Carlos A. Bacino, Anu Cherukuri, William R. Wilcox, Klaus Mohnike   +37 more
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Achondroplasia treatments in children aged 5 and older [PDF]

Molecular and Cellular Pediatrics
Achondroplasia, the most prevalent skeletal dysplasia, is a genetic disorder caused by activating mutations in the FGFR3 gene impairing endochondral ossification of long bones.
Emmanuelle Zakheim, Sonal Sachdeva, Daniel Moon, Miguel Nicole Ortiz, Nishant Mistry, Floyd Culler   +5 more
doaj   +3 more sources

Hypertrichosis associated with the use of C-type natriuretic peptide analogue: a case report [PDF]

Journal of Medical Case Reports
Background Achondroplasia is the most common form of disproportionate skeletal dysplasia. Recently, C-type natriuretic peptide analogue (vosoritide) was approved in numerous countries to increase annual growth velocity in patients with achondroplasia ...
Juan Llerena Clinton, Tatiana Sa Pacheco Carneiro Magalhaes, Ana Paula Bordallo, Paulo Ferrez Collett-Solberg   +3 more
doaj   +3 more sources

International expert opinion on the considerations for combining vosoritide and limb surgery: a modified delphi study [PDF]

Orphanet Journal of Rare Diseases
Background Achondroplasia is the most common form of skeletal disorder with disproportionate short stature. Vosoritide is the first disease-specific, precision pharmacotherapy to increase growth velocity in children with achondroplasia. Limb surgery is a
Silvio Boero, Julia Vodopiutz, Mohamad Maghnie, Josep M. de Bergua, Ignacio Ginebreda, Hiroshi Kitoh, Micha Langendörfer, Antonio Leiva-Gea, Jason Malone, Philip McClure, Gabriel T. Mindler, Dmitry Popkov, Robert Rodl, Pablo Rosselli, Fabio Verdoni, Viktor Vilenskii, Aaron J. Huser   +16 more
doaj   +3 more sources

Vosoritide: First Approval

Drugs, 2021
DeclarationsFunding The preparation of this review was not supported by any external funding.Authorship and Conflict of interest During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the ...
Sean Duggan (4873369)
core   +3 more sources

Vosoritide in achondroplasia: a profile of its use

Drugs & Therapy Perspectives, 2022
   Declarations Funding The preparation of this review was not supported by any external funding. Authorship and conflict of interest Young-A Heo, a salaried employee of Adis International Ltd/Springer Nature and an editor of Drugs & Therapy Perspectives,
Young-A Heo (5060039)
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A phase 3, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of vosoritide in children with hypochondroplasia: CANOPY HCH-3 study design [PDF]

Therapeutic Advances in Endocrinology and Metabolism
Background Hypochondroplasia is a skeletal dysplasia characterized by disproportionate short stature that is caused by gain-of-function variants in the fibroblast growth factor receptor 3 gene ( FGFR3 ), which negatively regulates endochondral bone ...
Andrew Dauber, Ravi Savarirayan, Ricki Carroll, Moira Cheung, Valérie Cormier-Daire, Mohamad Maghnie, Noriyuki Namba, Oliver Semler, Sajda Ghani, Ian Sabir, Roberto Bassi   +10 more
doaj   +2 more sources

Vosoritide Therapy in Children with Achondroplasia: Early Experience and Practical Considerations for Clinical Practice

Advances in Therapy, 2023
Introduction!#!Vosoritide is the first precision medical therapy approved to increase growth velocity in children with achondroplasia. Sharing early prescribing experiences across different regions could provide a framework for developing practical ...
Janet M. Legare, Cormier-Daire, Valérie, Maha Faden, Hartmann, Gabriele, Rohrer, Tilman R., Legare, Janet M., Michael B. Bober, Ekkehart Lausch, Mohnike, Klaus, Wilcox, William R., Travessa, André, Andre M. Travessa, Sérgio B. Sousa, Semler, Oliver, Huser, Aaron J., Lausch, Ekkehart, Aaron J. Huser, Valérie Cormier-Daire, Frank Rutsch, Sousa, Sérgio, Bober, Michael B., David Deyle, Sousa, Sérgio B., Julie Hoover-Fong, Oliver Semler, Legare, Janet, Deyle, David, Rutsch, Frank, White, Klane K., Verardo, Angela, Tilman R. Rohrer, Travessa, Andre M., Ricki Carroll, Hoover-Fong, Julie, Pamela Smith, Faden, Maha, Bober, Michael, Angela Verardo, Carroll, Ricki, Gabriele Hartmann, Klane K. White, William R. Wilcox, Smith, Pamela, Klaus Mohnike   +43 more
core   +4 more sources