Results 121 to 130 of about 534 (161)
Parents’ Experience of Administering Vosoritide: A Daily Injectable for Children With Achondroplasia
Advances in Therapy, 2023 Introduction: Vosoritide is the first approved pharmacological treatment for achondroplasia and is indicated for at-home injectable administration by a trained caregiver.
Klaus Mohnike +2 more
exaly +9 more sources
Vosoritide Treatment Accelerates Bone Growth in Children With Achondroplasia
Future Rare Diseases, 2021 Vosoritide is a drug developed for the treatment of achondroplasia and has demonstrated increases in the growth velocity of children with this condition.
Julie Hoover-Fong +2 more
exaly +4 more sources
Vosoritide Treatment for Children With Hypochondroplasia: A Phase 2 Trial
EClinicalMedicineBACKGROUND: Hypochondroplasia is a rare autosomal dominant skeletal dysplasia due to activating variants in . It presents with disproportionate short stature with a wide range of clinical severity.
Roopa Kanakatti Shankar +2 more
exaly +4 more sources
Nature Reviews EndocrinologyAchondroplasia is the most common genetic form of short-limbed skeletal dysplasia (dwarfism). Clinical manifestations and complications can affect individuals across the lifespan, including the need for adaptations for activities of daily living, which ...
Klaus Mohnike +2 more
exaly +4 more sources
Lancet, The, 2020 There are no effective therapies for achondroplasia. An open-label study suggested that vosoritide administration might increase growth velocity in children with achondroplasia. This phase 3 trial was designed to further assess these preliminary findings.
Paul Harmatz, Klane K White
exaly +2 more sources
MedBACKGROUND: Vosoritide is a C-type natriuretic peptide analog that addresses an underlying pathway causing reduced bone growth in achondroplasia. Understanding the vosoritide treatment effect requires evaluation over an extended duration and comparison ...
Paul Harmatz +2 more
exaly +3 more sources