Results 141 to 150 of about 335,224 (186)

Elosulfase alfa (BMN 110) for the treatment of mucopolysaccharidosis IVA (Morquio A Syndrome)

Expert Review of Clinical Pharmacology, 2016
C. Hendriksz
semanticscholar   +1 more source

Safety, immunogenicity, and clinical outcomes in patients with Morquio A syndrome participating in 2 sequential open-label studies of elosulfase alfa enzyme replacement therapy (MOR-002/MOR-100), representing 5 years of treatment.

Molecular Genetics and Metabolism, 2018
C. Hendriksz, S. Santra, Simon A. Jones, T. Geberhiwot, L. Jesaitis, B. Long, Yulan Qi, Sara M. Hawley, C. Decker   +8 more
semanticscholar   +1 more source

Novel data on growth phenotype and causative genotypes in 29 patients with Morquio (Morquio-Brailsford) syndrome from Central-Eastern Europe. [PDF]

J Appl Genet, 2019
Jezela-Stanek A, Różdżyńska-Świątkowska A, Kulpanovich A, Ciara E, Marucha J, Tylki-Szymańska A.   +5 more
europepmc   +1 more source

Hip Dysplasia in Mucopolysaccharidosis Type IVA (Morquio A Syndrome) Treated by Proximal Femoral Valgization Osteotomy: A Case Report. [PDF]

J Orthop Case Rep, 2018
Berger-Groch J, Rupprecht M, Stuecker R, Muschol N, Breyer SR.   +4 more
europepmc   +1 more source

Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study.

Molecular Genetics and Metabolism, 2017
C. Hendriksz, R. Parini, M. AlSayed, J. Raiman, R. Giugliani, John J. Mitchell, B. Burton, N. Guelbert, F. Stewart, D. Hughes, Robert A. Matousek, Sara M. Hawley, C. Decker, P. Harmatz   +13 more
semanticscholar   +1 more source

TAVI-in-TAVI in a patient with morquio syndrome: a case report. [PDF]

Eur Heart J Case Rep
Chin DXL, De Michele G, Cristofani D, De Felice F.   +3 more
europepmc   +1 more source

Craniovertebral Junction Compression in Patients With Morquio Syndrome: Case Series and Literature Review. [PDF]

Cureus
Rakshit P, Sadayandi Ramesh A, Gopikrishnan R, Mani MK, Sathia Prabhu A.   +4 more
europepmc   +1 more source

Heart and Cardiovascular Involvement in Patients with Mucopolysaccharidosis Type IVA (Morquio-A Syndrome). [PDF]

PLoS One, 2016
Kampmann C, Abu-Tair T, Gökce S, Lampe C, Reinke J, Mengel E, Hennermann JB, Wiethoff CM.   +7 more
europepmc   +1 more source

MCV/Q, Medical College of Virginia Quarterly, Vol. 13 No. 4 [PDF]

, 1977

core   +1 more source

Surgical Reconstruction for Severe Tracheal Obstruction in Morquio A Syndrome.

Annals of Thoracic Surgery, 2016
C. Pizarro, R. Davies, M. Theroux, E. Spurrier, L. Averill, S. Tomatsu   +5 more
semanticscholar   +1 more source