Results 101 to 110 of about 534 (161)

P208: Safety profile and adherence of vosoritide in young children with achondroplasia in Japan

Genetics in Medicine Open
Toshimi Michigami, Taichi Kitaoka, Jeanne Pimenta, Veronika Horvathova, Hirofumi Tokuoka, Shirou Matsumoto   +5 more
doaj   +1 more source

Response to letter to the editor for "Infigratinib low dose therapy is an effective strategy to treat hypochondroplasia". [PDF]

J Bone Miner Res
Legeai-Mallet L, Shah BP.
europepmc   +1 more source

The CNP analogue vosoritide mediates PDE2-sensitive anti-arrhythmogenic effects in mouse hearts with STZ-induced type 1 diabetes. [PDF]

Basic Res Cardiol
Firneburg R, Tergau K, Cachorro E, Schubert M, Dhara A, Luo X, Klapproth E, Guan K, El-Armouche A, Kämmerer S.   +9 more
europepmc   +1 more source

The CrescNet Registry Achondroplasia Module: Real-World Demographic Data and Clinical Outcomes in Untreated and Vosoritide-Treated Individuals.

Horm Res Paediatr
Mohnike K, Beger C, Gausche R, Hoyer-Kuhn H, Muschol N, Palm K, Rohrer TR, Sredkova M, Streata I, Wechsung K, Woelfle J, Zeman J, Eibenstein U, Pimenta JM, Reichert A, Pfäffle R.   +15 more
europepmc   +1 more source

Limb lengthening in achondroplasia: a cost-effectiveness analysis using a Markov model. [PDF]

Orphanet J Rare Dis
Roza Miguel PO, Leiva Gea A, Rabanal Rubio M, García-Rodríguez N, González Fidalgo E.   +4 more
europepmc   +1 more source

P131: Persistence of growth-promoting effects in infants and toddlers with achondroplasia: Results from a phase II extension study with vosoritide*

Genetics in Medicine Open
Ravi Savarirayan, William Wilcox, Paul Harmatz, John Phillips, III, Lynda Polgreen, Louise Tofts, Keiichi Ozono, Paul Arundel, Melita Irving, Carlos Bacino, Donald Basel, Ricki Carroll, Joel Charrow, Hiroshi Mochizuki, Yumiko Kotani, Howard Saal, Lingling Han, Andrea Low, Elena Fisheleva, Jonathan Day   +19 more
doaj   +1 more source

Case Report: Proportionate short stature in a three-generation family harboring <i>FGFR3</i> N540S: phenotypic expansion beyond hypochondroplasia and implications for genetic screening in idiopathic short stature. [PDF]

Front Endocrinol (Lausanne)
Zhang B, Wang X, Luo J, Gong C.
europepmc   +1 more source

P144: Persistence of growth-promoting effects in children with achondroplasia up to 7 years: Update from phase 2 extension study with vosoritide*

Genetics in Medicine Open
Julie Hoover-Fong, Melita Irving, Carlos Bacino, Joel Charrow, Carlos Prada, Valerie Cormier-Daire, Lynda Polgreen, Paul Harmatz, Sajda Ghani, Elena Fisheleva, Andrea Low, Jonathan Day, John Phillips, III, Ravi Savarirayan   +13 more
doaj   +1 more source

Real-world Outcome of Vosoritide Treatment in Children With Achondroplasia: A 12-month Retrospective Observational Study. [PDF]

J Endocr Soc
Reincke S, Semler O, Junghänel-Welzing S, Stasek S, Rehberg M, Pfeiffer E, Hoyer-Kuhn H.   +6 more
europepmc   +1 more source