Results 21 to 30 of about 676 (154)

Safety and Efficacy of Elosulfase Alfa in Australian Patients with Morquio a Syndrome: A Phase 3b Study [PDF]

Journal of Inborn Errors of Metabolism and Screening, 2020
The safety and efficacy of elosulfase alfa were evaluated in a multicenter, open-label, phase 3b study in Australian Morquio A patients, consisting of a 49-week initial phase and an extension phase until elosulfase alfa was government funded.
Kaustuv Bhattacharya, Shanti Balasubramaniam, Kevin Murray, Heidi Peters, David Ketteridge, Anita Inwood, Joy Lee, Carolyn Ellaway, Penny Owens, Melanie Wong, Christine Ly, Jim McGill   +11 more
doaj   +4 more sources

Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks [PDF]

Journal of Inborn Errors of Metabolism and Screening, 2017
Patients with mucopolysaccharidosis (MPS), and Morquio A syndrome (MPS IVA) in particular, often report substantial pain burden. MOR-008 was a randomized, double-blind, pilot study assessing the safety and efficacy, including impact on patient-reported ...
Marsha Treadwell, Paul R. Harmatz, Barbara K. Burton, John J. Mitchell, Nicole Muschol, Simon A. Jones, Gregory M. Pastores, Heather A. Lau, Rebecca Sparkes, V. Reid Sutton, Bianca Meesen, Christine A. Haller, Adam J. Shaywitz, Jeffrey I. Gold   +13 more
doaj   +5 more sources

Judicialization and right to health in Brazil: a trajectory of matches and mismatches. [PDF]

Rev Saude Publica, 2023
Neste texto, discutem-se os impactos da judicialização na garantia do direito à saúde no Brasil e a necessidade de reavaliação do papel do Judiciário na sua proteção. Evidências da literatura técnico-científica e informações sobre a execução orçamentário-
Vieira FS.
europepmc   +3 more sources

Consensus statement on enzyme replacement therapy for mucopolysaccharidosis IVA in Central and South-Eastern European countries [PDF]

Orphanet Journal of Rare Diseases, 2022
Background Mucopolysaccharidosis IVA (MPS IVA), or Morquio A syndrome, is a rare inherited metabolic disorder caused by deficiency of the lysosomal enzyme N-acetylgalactosamine-6-sulfatase.
Martin Magner, Zsuzsanna Almássy, Zoran Gucev, Beata Kieć-Wilk, Vasilica Plaiasu, Anna Tylki-Szymańska, Dimitrios Zafeiriou, Ioannis Zaganas, Christina Lampe   +8 more
doaj   +2 more sources

Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome [PDF]

Molecular Genetics and Metabolism, 2016
Long-term efficacy and safety of elosulfase alfa enzyme replacement therapy were evaluated in Morquio A patients over 96weeks (reaching 120weeks in total from pre-treatment baseline) in an open-label, multi-center, phase III extension study.
AlSayed, MD, Berger, KI, Burton, BK, Giugliani, R, Guelbert, N, Harmatz, PR, Hendriksz, CJ, Hughes, DA, Jurecki, E, Matousek, R, Mitchell, JJ, Parini, R, Raiman, J, Shaywitz, AJ, Slasor, P, Solano Villarreal, ML, Stewart, F   +16 more
core   +4 more sources

Diagnostic journey and impact of enzyme replacement therapy for mucopolysaccharidosis IVA: a sibling control study [PDF]

Orphanet Journal of Rare Diseases, 2020
Background Mucopolysaccharidosis (MPS) IVA, also known as Morquio A syndrome, is a rare autosomal recessive lysosomal storage disorder caused by a deficiency in the enzyme N-acetylgalactosamine-6-sulfatase.
Can Ficicioglu, Dena R. Matalon, Nicole Luongo, Caitlin Menello, Tracy Kornafel, Andrew J. Degnan   +5 more
doaj   +3 more sources

Elosulfase alfa (BMN 110) for the treatment of Mucopolysaccharidosis IVA (Morquio A syndrome) [PDF]

Expert Review of Clinical Pharmacology, 2016
INTRODUCTION : Morquio A syndrome is a rare, autosomal recessive, lysosomal storage disorder caused bya deficiency in the enzyme N-acetylgalactosamine-6-sulfatase (GALNS).
Hendriksz, Christian J.
core   +4 more sources

Long-term outcomes of enzyme replacement therapy from a large cohort of Korean patients with mucopolysaccharidosis IVA (Morquio A syndrome) [PDF]

Molecular Genetics and Metabolism Reports
Mucopolysaccharidosis (MPS) IVA (Morquio A syndrome) is an autosomal recessive lysosomal storage disorder caused by a mutation affecting the enzyme N-acetylgalactosamine-6-sulfatase (EC 3.1.6.4, GALNS).
Juyoung Sung, Insung Kim, Minji Im, Yoon Ji Ahn, Sang-Mi Kim, Ja-Hyun Jang, Hyung-Doo Park, Tae Yeon Jeon, Kyung Rae Ko, Se-Jun Park, Jun Hwa Lee, Eun Young Kim, Chong Kun Cheon, Eungu Kang, Jung-eun Moon, Young Bae Sohn, Hsiang-Yu Lin, Chih-Kuang Chuang, Shuan-Pei Lin, Sung Yoon Cho   +19 more
doaj   +2 more sources

Efficacy of different treatment strategies in patients with mucopolysaccharidosis: a systematic review and network meta-analysis of randomized controlled trials [PDF]

Orphanet Journal of Rare Diseases
This systematic review of randomized controlled trials (RCT) was conducted to evaluate the efficacy of enzyme replacement therapy (ERT) for patients with mucopolysaccharidosis (MPS). We systematically searched PubMed, Embase, Web of Science, and Cochrane
Lingling Huang, Jianru Wu, Biyu Tang, Jingying Wu, Fenfang Wei, Hong Qiao Li, Limin Li, Xinru Wang, Bei Wang, Wenyu Wu, Xiang Hong   +10 more
doaj   +2 more sources

Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis. [PDF]

J Pers Med, 2022
Mucopolysaccharidosis type IVA (MPS IVA or Morquio A), a lysosomal storage disease with an autosomal recessive inherited pattern, is induced by GALNS gene mutations causing deficiency in N-acetylgalactosamine-6-sulfatase activity (GALNS; EC 3.1.6.4).
Lee CL, Chuang CK, Syu YM, Chiu HC, Tu YR, Lo YT, Chang YH, Lin HY, Lin SP.   +8 more
europepmc   +3 more sources