Results 31 to 40 of about 534 (161)

Gait and posturographic parameters of children with achondroplasia – a long-term pilot study [PDF]

BMC Musculoskeletal Disorders
Walking and maintaining balance are essential for participating in daily life. For children with achondroplasia, reaching these milestones is with a delay and during growth, challenges persist for those skills.
Mareike Hergenröther, Katja Palm, Klaus Mohnike, Kerstin Witte   +3 more
doaj   +2 more sources

Clinical Significance of Drug–Drug Interaction Studies During Therapeutic Peptide Drug Development: Follow‐Up Investigation of Therapeutic Peptides Approved Between 2021 and 2024

Clinical and Translational Science
The risk of clinically relevant drug–drug interactions (DDIs) for therapeutic peptides remains unclear, mandating a comprehensive analysis for this modality.
Carolina Säll, Upendra Argikar, Constanze Hilgendorf, Hilmar Schiller, Anders Sonesson, Kenichi Umehara, Kai Wang   +6 more
doaj   +2 more sources

Advances in the treatment of achondroplasia [PDF]

Journal of Education, Health and Sport
Achondroplasia is a condition resulting from a missense mutation in the FGFR3 (fibroblast growth factor receptor 3) gene, representing the predominant etiology of short height in humans.
Patrycja Jędrzejewska-Rzezak
doaj   +2 more sources

Emerging NPR-B agonists for cartilage and bone: from CNP biology to translational compound design [PDF]

Frontiers in Chemistry
Disorders affecting cartilage and bone development frequently originate from disrupted endochondral ossification within growth-plate cartilage. Nonetheless, few therapeutic interventions directly target the local signaling pathways governing this process.
Yasuhiko Ago, Yasuhiko Ago, Amali Karunathilaka, Amali Karunathilaka, Jacky M. Guerrero-Vargas, Jacky M. Guerrero-Vargas, Shunji Tomatsu, Shunji Tomatsu   +7 more
doaj   +2 more sources

Development of Muscle Function in Children with Achondroplasia Under Vosoritide Treatment: A Retrospective Single-Centre Observational Study. [PDF]

J Musculoskelet Neuronal Interact
OBJECTIVES: Vosoritide, a C-type natriuretic peptide analog, is the first approved pharmacological treatment for achondroplasia that targets the overactive FGFR3 pathway and promotes longitudinal bone growth.
Reincke S, Stasek S, Junghänel-Welzing S, Semler O, Rehberg M, Duran I, Hoyer-Kuhn H.   +6 more
europepmc   +2 more sources

Real-World Safety and Effectiveness of Vosoritide in Children with Achondroplasia: French Early Access Program.

Horm Res Paediatr
International audienceIntroduction: Vosoritide is the first approved treatment for achondroplasia, a rare genetic disorder that results in disproportionate short stature.
Cormier-Daire V, Edouard T, Isidor B, Mukherjee S, Pimenta JM, Rossi M, Schaefer E, Sigaudy S, Baujat G.   +8 more
europepmc   +2 more sources

Current Strategies and Future Prospects of Achondroplasia Treatment : A Systemic Review [PDF]

Journal of Education, Health and Sport
Introduction and Purpose: Achondroplasia, the most common form of dwarfism, results from mutations in the FGFR3 gene. The disorder causes disproportionate short stature due to a gain-of-function mutation in FGFR3, impacting bone growth.
Daria Bednarczyk, Wiktoria Pysiewicz, Wiktor Garbarczyk, Agnieszka Napieralska, Albert Kapla, Michalina Jurkiewicz, Julia Białeta, Katarzyna Rowińska, Karolina Siembab, Alicja Černohorská   +9 more
doaj   +2 more sources

Persistent and Stable Growth Promoting Effects of Vosoritide in Children With Achondroplasia for up to 2 Years: Results From the Ongoing Phase 3 Extension Study [PDF]

, 2021
Objectives: Vosoritide is a potent stimulator of endochondral bone growth and is in development for the treatment of achondroplasia, the most common form of disproportionate short stature.
Tofts, Louise, Savarirayan, Ravi, Kagami, Shoji, Porco, Dania, Ginebreda, Ignacio, Ozono, Keiichi, Mohnike, Klaus, Charrow, Joel, Irving, Melita, Wilcox, William, Bober, Michael B, Jayaram, Kala, Polgreen, Lynda, Hoernschemeyer, Daniel, Arundel, Paul, White, Klane, Harmatz, Paul, Leiva-Gea, Antonio, Fisheleva, Elena, Rutsch, Frank, Yasui, Natsuo, Basel, Donald, Hoover-Fong, Julie, Alanay, Yasemin, Han, Lynn, Saal, Howard, Bacino, Carlos A, Mochizuki, Hiroshi, Luna-González, Felipe, Day, Jonathan, Font, Rosendo Ullot   +30 more
core   +3 more sources

Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study [PDF]

, 2021
PURPOSE: Achondroplasia is caused by pathogenic variants in the fibroblast growth factor receptor 3 gene that lead to impaired endochondral ossification.
Day, Jonathan R S, Luna-González, F, White, Klane K, Mohnike, Klaus, Charrow, Joel, Charrow, J, Kotani, Yumiko, Bober, MB, Saal, Howard M, Irving, M, Hoernschemeyer, Daniel, Arundel, Paul, Day, JRS, Porco, Dania M, Bacino, CA, Ginebreda, I, White, KK, Basel, D, Leiva-Gea, Antonio, Fisheleva, Elena, Basel, Donald, Hoover-Fong, J, Hoover-Fong, Julie, Alanay, Yasemin, Day, Jonathan RS, Alanay, Y, Polgreen, LE, Font, RU, Huntsman-Labed, A, Yasui, N, Wilcox, William R, Jayaram, K, Kotani, Y, Font, Rosendo Ullot, Arundel, P, Tofts, Louise, Savarirayan, Ravi, Ginebreda, Ignacio, Ozono, Keiichi, Hoernschemeyer, D, Fisheleva, E, Wilcox, WR, Huntsman-Labed, Alice, Polgreen, Lynda E, Irving, Melita, Ozono, K, Savarirayan, R, Bober, Michael B, Jayaram, Kala, Mohnike, K, Saal, HM, Harmatz, Paul, Rutsch, Frank, Yasui, Natsuo, Rutsch, F, Mochizuki, H, Harmatz, P, Leiva-Gea, A, Bacino, Carlos A, Tofts, L, Mochizuki, Hiroshi, Porco, DM, Luna-González, Felipe, Day, Jonathan R. S., Wilcox, William R., Porco, Dania M., White, Klane K., Polgreen, Lynda E., Bacino, Carlos A., Saal, Howard M., Bober, Michael B., Alice Huntsman-Labed, Paul Arundel, Dania M. Porco, Antonio Leiva-Gea, Jonathan R.S. Day, Frank Rutsch, Yumiko Kotani, Ravi Savarirayan, Klane K. White, Daniel Hoernschemeyer, Hiroshi Mochizuki, Natsuo Yasui, Joel Charrow, Felipe Luna-González, Melita Irving, Rosendo Ullot Font, William R. Wilcox, Klaus Mohnike, Donald Basel, Kala Jayaram, Michael B. Bober, Yasemin Alanay, Lynda E. Polgreen, Ignacio Ginebreda, Paul Harmatz, Julie Hoover-Fong, Keiichi Ozono, Louise Tofts, Elena Fisheleva, Howard M. Saal, Carlos A. Bacino   +101 more
core   +1 more source

Collagen-binding C-type natriuretic peptide enhances chondrogenesis and osteogenesis [PDF]

JCI Insight
C-type natriuretic peptide (CNP) is known to promote chondrocyte proliferation and bone formation; however, CNP’s extremely short half-life necessitates continuous intravascular administration to achieve bone-lengthening effects. Vosoritide, a CNP analog
Kenta Hirai, Kenta Sawamura, Ryusaku Esaki, Ryusuke Sawada, Yuka Okusha, Eriko Aoyama, Hiroki Saito, Kentaro Uchida, Takehiko Mima, Satoshi Kubota, Hirokazu Tsukahara, Shiro Imagama, Masaki Matsushita, Osamu Matsushita, Yasuyuki Hosono   +14 more
doaj   +2 more sources