Results 111 to 120 of about 1,275 (171)

Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study. [PDF]

Mol Genet Metab, 2022
Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D, HGT-HIT-094 Study Group.   +14 more
europepmc   +1 more source

Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II. [PDF]

Mol Genet Metab, 2022
Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D, SHP609-302 study group.   +14 more
europepmc   +1 more source

Successful Idursulfase Desensitization Experience in a Pediatric Patient

Asthma Allergy Immunology, 2022
Zeynep ŞENGÜL EMEKSIZ, Şule BÜYÜK YAYTOKGIL, Ilknur KÜLHAŞ ÇELIK, Çiğdem KASAPKARA, Emine DIBEK MISIRLIOĞLU   +4 more
openaire   +1 more source

Carpal Tunnel Syndrome Attributed to Medication Use: A Pharmacovigilance Study. [PDF]

Cureus
Mihalache A, Volfson E, Huang R, Zuo K, Persitz J.   +4 more
europepmc   +1 more source

Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey. [PDF]

Orphanet J Rare Dis
Muenzer J, Amartino H, Giugliani R, Harmatz P, Lin SP, Link B, Molter D, Ramaswami U, Scarpa M, Botha J, Audi J, Burton BK.   +11 more
europepmc   +1 more source

Drug delivery related to metabolites in human breath – first results of long term clinical studies using MCC/IMS [PDF]

, 2011
Baumbach, Jörg Ingo, Litterst, P., Maddula, Sasidhar, Westhoff, M.   +3 more
core  

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

Orphanet J Rare Dis
Huang L, Wu J, Tang B, Wu J, Wei F, Li HQ, Li L, Wang X, Wang B, Wu W, Hong X.   +10 more
europepmc   +1 more source

Evidence and recommendation for mucopolysaccharidosis type II newborn screening in the United States. [PDF]

Genet Med, 2023
Ream MA, Lam WKK, Grosse SD, Ojodu J, Jones E, Prosser LA, Rosé AM, Comeau AM, Tanksley S, Powell CM, Kemper AR.   +10 more
europepmc   +1 more source

Potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan drugs [PDF]

, 2016

core   +1 more source

Intravenous Idursulfase for the Treatment of Mucopolysaccharidosis Type II: A Systematic Literature Review. [PDF]

Int J Mol Sci
Al-Hertani W, Pathak RR, Evuarherhe O, Carter G, Schaeffer-Koziol CR, Whiteman DAH, Wright E.   +6 more
europepmc   +1 more source