Results 91 to 100 of about 172,212 (109)

Innovative Mechanisms of Pre-Marketing Authorization Access for Rare Diseases in Brazil: A Case Study of Pabinafusp-Alfa for Mucopolysaccharidosis Type II

Jornal Brasileiro De Economia Da Saúde, 2023
Patients with rare diseases frequently face unmet medical needs due to the high costs, lengthy development times, and slow approval processes for new treatments. This case study discusses innovative access alternatives for rare diseases in Brazil, focusing on early access to pabinafusp-alfa for mucopolysaccharidosis type II (MPS-II), a rare genetic ...
Maicon Falavigna, Celina Migliavaca, Elio Tanaka, Marcos Santos   +3 more
exaly   +2 more sources

The Impact of Pabinafusp Alfa on the Disease Burden in Hunter's Syndrome: Patient-Reported Outcomes

Journal of Pediatric Neurology
AbstractSevere mucopolysaccharidosis type II (MPS-II) patients present with progressive mental impairment and reduced life expectancy. While current available treatment cannot cross the blood–brain barrier, the enzyme replacement therapy with pabinafusp alfa (a recombinant iduronate-2-sulfatase fused to an antihuman transferrin receptor antibody) was ...
Roberto Giugliani, Carolina Fischinger Moura de Souza, Edna Tiemi Sakata   +2 more
exaly   +2 more sources

Real-World Data of Enzyme Replacement Therapy With Pabinafusp Alfa for Neuronopathic MPS-II: Updated Clinical Data From Japan

Molecular Genetics and Metabolism, 2023
Yuji Sato, Norio Sakai, Kazunori Tanizawa   +2 more
exaly   +2 more sources

Intravenous Treatment With Pabinafusp Alfa Dose-Dependently Prevents Neurological Impairment and Bone Deformities in a Mouse Model of Mucopolysaccharidosis Type II

Molecular Genetics and Metabolism, 2023
Kohtaro Minami, Hideto Morimoto, Tohru Hirato   +2 more
exaly   +2 more sources

Long-Term Neurodevelopmental Changes in Subjects With MPS II Following Long-Term Treatment With Pabinafusp Alfa: An Integrated Analysis From Pre- and Post-Approval Clinical Trials in Brazil and Japan

Molecular Genetics and Metabolism, 2023
Kazunori Tanizawa, Sairei So, Mathias Schmidt   +2 more
exaly   +2 more sources

Recovery of Retinal Function in MPS II Mice by Treatment With Pabinafusp Alfa

Molecular Genetics and Metabolism
Hideto Morimoto, Hiroyuki Sonoda, Eiji Yoden   +2 more
exaly   +2 more sources

Infusion Rate Adjustment in Enzyme Replacement Therapy With Pabinafusp Alfa for Mucopolysaccharidosis Type II

Molecular Genetics and Metabolism
Yuji Sato, Norio Sakai, Tatsuyoshi Yamamoto   +2 more
exaly   +2 more sources

A Global Phase III Study of Pabinafusp Alfa (JR-141) for Neuronopathic Mucopolysaccharidosis Type II: Updated Study Design

Molecular Genetics and Metabolism
Paul Harmatz, Yuji Sato, Kazunori Tanizawa   +2 more
exaly   +2 more sources

Long-Term Somatic Efficacy of Pabinafusp Alfa Across a Broad Spectrum of Age Groups and Phenotypes in Patients With Mucopolysaccharidosis Type II

Molecular Genetics and Metabolism
Motomichi Kosuga, Takashi Hamazaki, Toshiaki Ikeda   +2 more
exaly   +2 more sources

Sustained Cognitive and Adaptive Behavior Outcomes of Long − Term Treatment With Pabinafusp Alfa in Patients With Severe or Attenuated Mucopolysaccharidosis Type II

Molecular Genetics and Metabolism
Motomichi Kosuga, Takashi Hamazaki, Toshiaki Ikeda   +2 more
exaly   +2 more sources