Results 91 to 100 of about 534 (161)

Achondroplasia: Current Disease-Modifying Therapies and Genome Editing Strategies. [PDF]

Int J Mol Sci
Kondrateva E, Smirnikhina S.
europepmc   +1 more source

Real-world growth outcomes of vosoritide in children with achondroplasia: a single-center cohort study. [PDF]

J Endocr Soc
Regev R, Waksman Y, Cohen-Sela E, Brener A, Dechovich N, Gal M, Borger O, Zeitlin L, Lebenthal Y.   +8 more
europepmc   +1 more source

Publisher Correction: 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

A phase II basket trial of vosoritide in children with RASopathies, ACAN, and NPR2 deficiency.

J Clin Endocrinol Metab
Dauber A, Zhang A, Shafaei N, Seaforth R, Pitner K, Dham N, Kanakatti Shankar R, Merchant N.   +7 more
europepmc   +1 more source

P354: Vosoritide as a targeted therapy for FGFR3-related thanatophoric dysplasia

Genetics in Medicine Open
Benjamin Cocanougher, Rose Sharpe, Mariele Anneling, Brooke Baker, Nicole Panosh, Dmitriy Niyazov, Susan Izatt, Jennifer Cohen   +7 more
doaj   +1 more source

Healthcare and Psychosocial Needs in Achondroplasia Across the Lifespan: Developmental Functioning, Multidisciplinary Care, and Family-Centered Outcomes. [PDF]

Healthcare (Basel)
Șerban RC, Mitut-Veliscu A, Dumitra A, Marica L, Popescu C, Costache A, Teona Ș, Riza AL, Dirnu R, Pluta ID, Varut RM, Streata I.   +11 more
europepmc   +1 more source

Long-term vosoritide treatment sustains bone growth and may enhance wellbeing in children with achondroplasia: a plain language summary

Future Rare Diseases
Florian Innig, Victoria Garcia, Marco Sessa, Marco Stadler, Julie Hoover-Fong   +4 more
doaj   +1 more source

O03: Vosoritide increases growth velocity in hypochondroplasia: Phase 2 trial results

Genetics in Medicine Open
Andrew Dauber, Anqing Zhang, Roopa Kanakatti Shankar, Kimberly Boucher, Tara McCarthy, Niusha Shafaei, Raheem Seaforth, Meryll Castro, Niti Dham, Nadia Merchant   +9 more
doaj   +1 more source

A Randomized Controlled Trial of Vosoritide in Children with Achondroplasia

, 2020
Savarirayan, R, Tofts, L, Irving, M, Wilcox, W, Bacino, C, Hoover-Fong, J, Ullot R, Harmatz, P, Rutsch, F, Bober, M, Polgreen, L, Ginebreda, I, Mohnike, K, Charrow, J, Hoernschmeyer, D, Ozono, K, Alanay, Y, Arundel, P, Kagami, S, Yasui, N, White, K, Saal, H, Leiva-Gea, A, Luna-Gonzalez, F, Mochizuki, H, Basel, D, Porco, D, Jayaram, K, Fisheleva, E, Huntsman-Labed, A, Day, J   +30 more
openaire   +1 more source

Combination therapy with growth hormone and vosoritide for achondroplasia

Clinical Pediatric Endocrinology
Daisuke Harada, Kaoru Ueyama, Yoshiki Seino, Hiroko Kashiwagi   +3 more
openaire   +1 more source