Antibodies that neutralize cellular uptake of elosulfase alfa are not associated with reduced efficacy or pharmacodynamic effect in individuals with Morquio A syndrome.
Sign Up to like & getrecommendations! Published in 2017 at "Journal of immunological methods"
DOI: 10.1016/j.jim.2016.10.006
Abstract: Many enzyme replacement therapies (ERTs) for lysosomal storage disorders use the cell-surface cation-independent mannose-6 phosphate receptor (CI-M6PR) to deliver ERTs to the lysosome. However, neutralizing antibodies (NAb) may interfere with this process. We previously reported… read more here.
Keywords: antibodies neutralize; individuals morquio; efficacy; neutralize cellular ... See more keywords
Elosulfase alfa for mucopolysaccharidosis type IVA: Real-world experience in 7 patients from the Spanish Morquio-A early access program
Sign Up to like & getrecommendations! Published in 2018 at "Molecular Genetics and Metabolism Reports"
DOI: 10.1016/j.ymgmr.2018.03.009
Abstract: There is a growing interest in evaluating the effectiveness of enzyme replacement therapy (ERT) with elosulfase alfa in patients with mucopolysaccharidosis type IVA (MPS-IVA) under real-world conditions. We present the experience of seven pediatric MPS-IVA… read more here.
Keywords: iva real; mucopolysaccharidosis type; real world; type iva ... See more keywords
Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England
Sign Up to like & getrecommendations! Published in 2021 at "Orphanet Journal of Rare Diseases"
DOI: 10.1186/s13023-021-01675-x
Abstract: Background We present baseline characteristics and follow-up data of a Managed Access Agreement (MAA), including patients with mucopolysaccharidosis IVA (MPS IVA) receiving elosulfase alfa enzyme replacement therapy (ERT) in England on a conditional basis. Patients… read more here.
Keywords: access agreement; patients mucopolysaccharidosis; treatment; clinical patient ... See more keywords
Successful Elosulfase Alfa Desensitization Protocol in a Patient With Morquio A Syndrome.
Sign Up to like & getrecommendations! Published in 2022 at "Pediatrics"
DOI: 10.1542/peds.2021-052648
Abstract: Patients with lysosomal storage diseases may require modifications to standard drug desensitization protocols; personalized medicine as well as development of new treatment options are needed. read more here.
Keywords: desensitization; successful elosulfase; elosulfase alfa; alfa desensitization ... See more keywords