Vosoritide Research

Kunkel P, Vleggeert-Lankamp C, Riganti S, Leiva-Gea A, Boero S, Fredwall S et al.

Achondroplasia is associated with multisystem impairments that may require surgical interventions associated with high complication rates. The orthopaedic treatment landscape in achondroplasia varies globally, with some countries recommending surgical intervention more commonly. Achondroplasia-sp...

Ping H, Ding R, Huang C, Peng Y, Zhong Z, Wang W et al.

Achondroplasia (ACH) is a common inherited skeletal dysplasia (inherited dwarfism) that compromises quality of life across the lifespan. In 2021, vosoritide became the first approved precision therapy for ACH and is now available in more than 40 countries. Compared with prior symptomatic measures...

Mohnike K, Beger C, Gausche R, Hoyer-Kuhn H, Muschol N, Palm K et al.

Achondroplasia is a rare skeletal dysplasia characterized by severe disproportionate short stature. Vosoritide is currently the only approved therapy. The CrescNet registry is a network of primary and specialized pediatric tertiary centers that aims to improve early detection of growth disorders ...

Li X, Luo X, Liu F, Wang C, Li X, Yu H et al.

Dwarfism, a condition characterized by short stature, has been the focus of therapeutic advancements with the emergence of novel peptide drugs. Vosoritide, indicated for certain types of dwarfism, has shown therapeutic potential in clinical trials. However, a comprehensive safety profile is essen...

Singerline H, Laufman J, Wallis K, Merrill M

Homozygous achondroplasia is widely considered perinatal lethal by the medical community. In this case series, we report two children from a single family with longer-term survival. One child lived for 17 months and the other was 60 months at the time of publication. We describe two...

Hirai K, Sawamura K, Esaki R, Sawada R, Okusha Y, Aoyama E et al.

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 designed for resistance to neutral endopep...

Albuquerque ALB, Dacoregio MI, Rodrigues CG, Bertola DR, Zattar Ribeiro PV

Achondroplasia is the most common skeletal dysplasia, caused by gain-of-function variants in FGFR3, resulting in constitutive receptor activation and downstream inhibition of endochondral ossification. In 2021, the first targeted therapy, vosoritide, was approved in some countries after a landmar...

Papadimitriou DT

Children with craniopharyngioma exhibit a notably high incidence of normal-accelerated growth before and/or after neurosurgery/hypophysectomy despite confirmed panhypopituitarism, this constituting what is known as the growth without GH syndrome, which, to date, remains an unsolved mystery. Most ...

Hoskens H, Aponte JD, Da Silva C, Coward T, Hallgrímsson B, Irving M et al.

Achondroplasia is the most common form of disproportionate short-stature skeletal dysplasia. Constitutively activated FGFR3 signaling disrupts endochondral ossification, affecting long bone growth, as well as the craniofacial skeleton and skull base. This results in the distinctive craniofacial f...

Ireland PJ, Carroll T, Cotton Z, Ludwig K, Weston E, Munns CF et al.

Shimada T, Nakayama H, Ueda I, Ishimi T, Yamada C, Nakano Y et al.

Achondroplasia (ACH), a skeletal dysplasia, is characterized by disproportionate short stature and impaired quality of life. In Japan, growth hormone (GH) therapy has been available since 1997, and vosoritide, a C-type natriuretic peptide analog, was approved in 2022. Although clinical trials hav...

Kanakatti Shankar R, Galetaki DM, Zhang A, Shafaei N, Pitner K, Seaforth R et al.

Insulin-like growth factor-1 (IGF-1) and C-type natriuretic peptide (CNP) promote endochondral bone growth. We previously reported that vosoritide, a CNP analog, increases annualized growth velocity (AGV) and height standard deviation (SD) in pre-pubertal children with hypochondroplasia (HCH).We ...

Zakheim E, Sachdeva S, Moon D, Ortiz MN, Mistry N, Culler F et al.

Achondroplasia, the most prevalent skeletal dysplasia, is a genetic disorder caused by activating mutations in the FGFR3 gene impairing endochondral ossification of long bones. Clinical manifestations include disproportionate short stature and multisystem complications. Management has been limite...

Montero-Lopez R, Blaschitz A, Karas K, Fritz T, Laurer E, Naidoo U et al.

Thanatophoric dysplasia type 1 (TD1) is the most severe form of FGFR3-related skeletal dysplasia, with high perinatal mortality and no approved pharmacologic therapies. Vosoritide, a C-type natriuretic peptide analogue that counteracts FGFR3 overactivation, improves growth in achondroplasia, but ...

Firneburg R, Tergau K, Cachorro E, Schubert M, Dhara A, Luo X et al.

Diabetes mellitus induces adverse structural, electrophysiological and autonomic remodelling increasing the risk for life-threatening arrhythmias, particularly after acute myocardial infarction. Natriuretic peptides (NPs) show increasing evidence of antagonising arrhythmia. Our previous study dem...

Jones HL, Nania TI, Moore JM, Reed JD, Lyons AW, Potter P et al.

Achondroplasia is the most common genetic skeletal dysplasia, caused by activating mutations in the FGFR3 gene that impair endochondral ossification and result in disproportionate short stature. Vosoritide (VOXZOGO®), a C-type natriuretic peptide analog, is the first targeted therapy approve...

Bustamante Velez VH, Dowlut-McElroy T, Kanakatti Shankar R

Turner Syndrome (TS) is a genetic condition often characterized by ovarian insufficiency and infertility. Fertility preservation discussions are recommended early in care, but few studies have explored parental perspectives. Our objective was to assess the knowledge, perceptions, and attitudes re...

Allegri AEM, Bedeschi MF, Bocchi MB, Camurri V, Gonfiantini MV, Leoni C et al.

Achondroplasia is the most common form of disproportionate short stature and can lead to serious medical complications, including foramen magnum and spinal stenosis. Until 2021, there were no precision treatments available, and in some countries, elective surgery was considered a standard approac...