Results 91 to 100 of about 31,866 (306)

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study. [PDF]

, 2014
ObjectiveTo assess the efficacy and safety of enzyme replacement therapy (ERT) with BMN 110 (elosulfase alfa) in patients with Morquio A syndrome (mucopolysaccharidosis IVA).MethodsPatients with Morquio A aged ≥5 years (N = 176) were randomised (1:1:
A McDonald, AE Lammers, AM Li, AM Montaño, American Thoracic Society, American Thoracic Society, Barbara Burton, C Hendriksz, Christian J. Hendriksz, CJ Hendriksz, DB Rubin, Debra Lounsbury, Derralynn Hughes, E Yasuda, Eugen Mengel, J Muenzer, J Muenzer, J Nelson, J Nelson, JE Wraith, LA Clarke, LA Martell, M Dvorak-Ewell, Maurizio Scarpa, MF Algahim, P Harmatz, P Harmatz, P Harmatz, P Harmatz, Paul Harmatz, Peter Slasor, R Aslam, RJ Barst, RJA Butland, Roberto Giugliani, S Tomatsu, Shuan-Pei Lin, Simon A. Jones, Thomas R. Fleming, TR Fleming, Vassili Valayannopoulos, VC Dũng, Wolfgang Dummer, Y Hochberg   +43 more
core   +2 more sources

Inflammation and mechanical force‐induced bone remodeling

Periodontology 2000, EarlyView.
Abstract Periodontitis arises from imbalanced host–microbe interactions, leading to dysbiosis and destructive inflammation. The host's innate and adaptive immune responses produce pro‐inflammatory mediators that stimulate destructive events, which cause loss of alveolar bone and connective tissue attachment.
Hyeran Helen Jeon, Xin Huang, Leticia Rojas Cortez, Puttipong Sripinun, Jung‐me Lee, Julie J. Hong, Dana T. Graves   +6 more
wiley   +1 more source

Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance. [PDF]

, 2019
IntroductionMucopolysaccharidosis (MPS) VI or Maroteaux-Lamy syndrome (253200) is an autosomal recessive lysosomal storage disorder caused by deficiency in N-acetylgalactosamine-4-sulfatase (arylsulfatase B).
Akyol, Mehmet Umut, Alden, Tord D, Amartino, Hernan, Ashworth, Jane, Belani, Kumar, Berger, Kenneth I, Borgo, Andrea, Braunlin, Elizabeth, Eto, Yoshikatsu, Giugliani, Roberto, Gold, Jeffrey I, Harmatz, Paul, Hendriksz, Christian, Jester, Andrea, Jones, Simon A, Karsli, Cengiz, Mackenzie, William, Marinho, Diane Ruschel, McFadyen, Andrew, McGill, Jim, Mitchell, John J, MPS Consensus Programme Co-Chairs, MPS Consensus Programme Steering Committee, Muenzer, Joseph, Okuyama, Torayuki, Orchard, Paul J, Scarpa, Maurizio, Stevens, Bob, Thomas, Sophie, Walker, Robert, Wynn, Robert   +30 more
core  

GBM heterogeneity as a function of variable epidermal growth factor receptor variant III activity. [PDF]

, 2016
Abnormal activation of the epidermal growth factor receptor (EGFR) due to a deletion of exons 2-7 of EGFR (EGFRvIII) is a common alteration in glioblastoma (GBM). While this alteration can drive gliomagenesis, tumors harboring EGFRvIII are heterogeneous.
David James, C, Engler, Jane R, Ewald, Andrew J, Gong, Henry, Huillard, Emmanuelle, Koshkakaryan, Gayane, Lindberg, Olle R, McKinney, Andrew, Molinaro, Annette M, Phillips, Joanna J, Robinson, Aaron E, Shieh, Joseph T   +11 more
core   +1 more source

Mapping molluscan endocrinology: a systematic and critical appraisal

Biological Reviews, Volume 101, Issue 2, Page 970-1002, April 2026.
ABSTRACT Historically, a vertebrate‐centric paradigm has framed our interpretation of molluscan endocrinology, with considerable research focusing on vertebrate‐type steroid hormones (e.g. oestrogens, testosterone). However, contradictory evidence on the occurrence of vertebrate‐type steroid hormones in molluscan tissues, and a lack of the specific ...
Konstantinos Panagiotidis, Thomas H. Miller, Olwenn V. Martin, Alice Baynes   +3 more
wiley   +1 more source

Anaerobic Degradation of Sulfated Polysaccharides by Two Novel Kiritimatiellales Strains Isolated From Black Sea Sediment

Frontiers in Microbiology, 2019
The marine environment contains a large diversity of sulfated polysaccharides and other glycopolymers. Saccharolytic microorganisms degrade these compounds through hydrolysis, which includes the hydrolysis of sulfate groups from sugars by sulfatases ...
Daan M. van Vliet, Susakul Palakawong Na Ayudthaya, Susakul Palakawong Na Ayudthaya, Sally Diop, Laura Villanueva, Alfons J. M. Stams, Alfons J. M. Stams, Irene Sánchez-Andrea   +7 more
doaj   +1 more source

Quantification of Single‐Cell Cysteine Using an Electrochemical Nanosensor for Predicting Tumor Disulfidptosis Susceptibility

Advanced Science, Volume 13, Issue 13, 3 March 2026.
Cysteine attracts interest due to its unique electrochemical activity and emerging relationship with disulfidptosis. This study develops a new electrochemical nanosensor, which represents the first of its kind capable of detecting intracellular cysteine for various types of tumor cells and primary tumor cells in mice to evaluate disulfidptosis ...
Congcong Zhang, Xiangdi Zhang, Shushen Li, Yuyang Li, Bei Yuan, Mingshuang Zheng, Shuo Zhang, Fangping Yuan, Min Jia, Lixia Lu, Jun Zhou, Zhenguo Zhang, Xin Du   +12 more
wiley   +1 more source

A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA.

Molecular Genetics and Metabolism, 2016
OBJECTIVE This was an open-label, phase 1/2 dose-escalation, safety trial of intrathecal recombinant human heparan-N-sulfatase (rhHNS) administered via intrathecal drug delivery device (IDDD) for treating mucopolysaccharidosis IIIA (NCT01155778). STUDY
Simon A. Jones, C. Breen, Fiona Heap, S. Rust, J. de Ruijter, E. Tump, J. P. Marchal, Luying Pan, Yongchang Qiu, Jou-Ku Chung, N. Nair, P. Haslett, A. Barbier, F. Wijburg   +13 more
semanticscholar   +1 more source

Gene identification for the cblD defect of vitamin B12 metabolism [PDF]

, 2008
Background Vitamin B12 (cobalamin) is an essential cofactor in several metabolic pathways. Intracellular conversion of cobalamin to its two coenzymes, adenosylcobalamin in mitochondria and methylcobalamin in the cytoplasm, is necessary for the ...
Baumgartner, M R, Coelho, D, Fowler, B, Lerner-Ellis, J P, Newbold, R F, Rosenblatt, D S, Stucki, M, Suormala, T   +7 more
core   +1 more source

Intrathecal idursulfase‐IT in children younger than 3 years with neuronopathic mucopolysaccharidosis II in a single‐arm, open‐label, phase 2/3 substudy and extension

JIMD Reports, Volume 67, Issue 2, March 2026.
Abstract Data from a phase 2/3, randomized, controlled, open‐label, multicenter trial in children with neuronopathic mucopolysaccharidosis II (MPS II; Hunter syndrome) older than 3 years suggested a benefit of intrathecal idursulfase‐IT on cognitive functioning in some patients. We describe a separate, parallel, open‐label, single‐arm, 52‐week substudy
Joseph Muenzer, Barbara K. Burton, Paul Harmatz, Luis González Gutiérrez‐Solana, Matilde Ruiz‐Garcia, Simon A. Jones, Nathalie Guffon, Michal Inbar‐Feigenberg, Drago Bratkovic, Stewart Rust, Michael Hale, Yuna Wu, Karen S. Yee, David A. H. Whiteman, David Alexanderian   +14 more
wiley   +1 more source