Results 101 to 109 of about 172,212 (109)

Integrated Long-Term Efficacy and Safety Data on Enzyme Replacement Therapy With Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis Type II (MPS II): Updated Clinical Data From Japan and Brazil

Molecular Genetics and Metabolism
Kazunori Tanizawa, Sairei So, Mathias Schmidt   +2 more
exaly   +2 more sources

Behavioral improvement in a 9-year-old patient with MPS II undergoing enzyme replacement therapy with pabinafusp alfa: A case report

Molecular Genetics and Metabolism, 2022
Carolina Souza, Ana Paula K. Boer, Luis A. Matuoka, Elaine C. Migliori, Otávio R. Costa, Marina B. Zambrano, Roberto Giugliani   +6 more
openaire   +1 more source

Reduction of heparan sulfate in the brain by pabinafusp alfa results in prevention of neurodegeneration and neurocognitive impairment in a mouse model of mucopolysaccharidosis type II

Molecular Genetics and Metabolism, 2021
Hideto Morimoto, Sachiho Kida, Eiji Yoden, Masafumi Kinoshita, Noboru Tanaka, Ryuji Yamamoto, Yuri Koshimura, Haruna Takagi, Kenichi Takahashi, Tohru Hirato, Kohtaro Minami, Hiroyuki Sonoda   +11 more
openaire   +1 more source

Exploration of the efficacy of pabinafusp-alfa (JR-141) on neurocognitive development in Hunter syndrome (MPS II): 52-week data from clinical trials in Japan and Brazil

Molecular Genetics and Metabolism, 2021
Roberto Giugliani, Ana Maria Martins, Torayuki Okuyama, Yoshikatsu Eto, Norio Sakai, Kimitoshi Nakamura, Sairei So, Tatsuyoshi Yamamoto, Mariko Yamaoka, Toshiaki Ikeda, Kazunori Tanizawa, Hiroyuki Sonoda, Mathias Schmidt, Yuji Sato   +13 more
openaire   +1 more source

Long term efficacy and safety of pabinafusp-alfa (JR-141) in Hunter syndrome (MPS-II): 104-week data from the clinical trials in Japan and Brazil

Molecular Genetics and Metabolism, 2022
Roberto Giugliani, Ana M. Martins, Torayuki Okuyama, Yoshikatsu Eto, Norio Sakai, Kimitoshi Nakamura, Sairei So, Tatsuyoshi Yamamoto, Mariko Yamaoka, Toshiaki Ikeda, Hiroaki Moriuchi, Kazunori Tanizawa, Hiroyuki Sonoda, Mathias Schmidt, Yuji Sato   +14 more
openaire   +1 more source

EE574 Innovative Mechanisms of Pre-Market Authorization Access for Rare Diseases in Brazil. a Case Study of Pabinafusp-Alfa for Mucopolysaccharidosis Type II

Value in Health, 2023
E. Tanaka, M. Falavigna, M.H. Romcy, G. Priszkulnik, M. Santos, A. Daher, E. Maia   +6 more
openaire   +1 more source

Changes in quality of life reflecting neurobehavioral improvements observed by caregivers/physicians of patients with neuronopathic mucopolysaccharidosis: An interview-based survey from Brazil following clinical trials with pabinafusp alfa

Molecular Genetics and Metabolism, 2023
Ana Maria Martins, Carmen Silvia Curiati Mendes, Marco A. Curiati, Marcelo H. Yamamoto, Ricardo Cunegundes, Sairei So, Kazunori Tanizawa, Hiroyuki Sonoda, Mathias Schmidt, Yuji Sato   +9 more
openaire   +1 more source

New product intros (diclofenac etalhyaluronate, emitasvir phosphate, (18F)fluoroestradiol, furmonertinib mesilate, idecabtagene vicleucel, lisocabtagene maraleucel, naxitamab, pabinafusp alfa, pamiparib, piflufolastat F18, rilonacept (new indication), sotrovimab, viloxazine hydrochloride (new indication/new formulation))

Drugs of Today, 2021
openaire   +1 more source

Long-term impact of pabinafusp alfa on disease burden in Hunter syndrome: A 4-year follow-up of patient-reported outcomes

Molecular Genetics and Metabolism
Ana Maria Martins, Carmen Silvia Curiati Mendes, Edna T. Sakata, Marco A. Curiati, Felippe R.E.O. Previdi, Jessica Espolaor, Marcelo H. Yamamoto, Yuji Sato, Ricardo Cunegundes   +8 more
openaire   +1 more source