BioMarin (United Kingdom)

C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia

Ravi Savarirayan, Melita Irving, Carlos A. Bacino et al. · New England Journal of Medicine · 2019 · 205 citations · Full text

Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial

Ravi Savarirayan, Louise Tofts, Melita Irving et al. · The Lancet · 2020 · 184 citations

Subcutaneous Vosoritide Therapy, Pediatrics, Multicentre Trial +2

Burden of disease in patients with Morquio A syndrome: results from an international patient-reported outcomes survey

Christian J. Hendriksz, Christine Lavery, Mahmut Çöker et al. · Orphanet Journal of Rare Diseases · 2014 · 92 citations · Full text

Clinical burden of illness in patients with phenylketonuria (PKU) and associated comorbidities - a retrospective study of German health insurance claims data

K. F. Trefz, Ania C. Muntau, KM Kohlscheen et al. · Orphanet Journal of Rare Diseases · 2019 · 89 citations · Full text

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

Ravi Savarirayan, Louise Tofts, Melita Irving et al. · Genetics in Medicine · 2021 · 88 citations · Full text