Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I

Ultrasound findings of finger, wrist and knee joints in Mucopolysaccharidosis Type I

10.1016/j.ymgme.2021.05.009 ◽

2021 ◽

Author(s):

Johannes Roth ◽

Michal Inbar-Feigenberg ◽

Julian Raiman ◽

Marg Bisch ◽

Pranesh Chakraborty ◽

...

Keyword(s):

Knee Joints ◽

Type I ◽

Ultrasound Findings

Phase I/II clinical trial design for a novel therapy for mucopolysaccharidosis type I with an intravenously administered blood-brain barrier-crossing enzyme (JR-171)

10.1016/j.ymgme.2020.12.105 ◽

2021 ◽

Vol 132 (2) ◽

pp. S49

Author(s):

Ryo Higurashi ◽

Mika Okazaki ◽

Satoshi Kawashima ◽

Toshiaki Ikeda ◽

Sairei So ◽

...

Keyword(s):

Clinical Trial ◽

Phase I ◽

Blood Brain Barrier ◽

Trial Design ◽

Clinical Trial Design ◽

Brain Barrier ◽

Type I ◽

Novel Therapy

Sexual behaviour in a murine model of mucopolysaccharidosis type I (MPS I)

PLoS ONE ◽

10.1371/journal.pone.0220429 ◽

2019 ◽

Vol 14 (12) ◽

pp. e0220429 ◽

Cited By ~ 1

Author(s):

Ana Barbosa Mendes ◽

Cinthia Castro do Nascimento ◽

Vânia D’Almeida

Keyword(s):

Sexual Behaviour ◽

Murine Model ◽

Type I ◽

Mps I

430. Antibodies to Recombinant Human α-L-Iduronidase Enhance Uptake into Macrophages in Murine Mucopolysaccharidosis Type I

Molecular Therapy ◽

10.1016/s1525-0016(16)33239-7 ◽

2016 ◽

Vol 24 ◽

pp. S170-S171

Author(s):

Valentina Sanghez ◽

Shih-Hsin Kan ◽

Steven Q Le ◽

Don Clarke ◽

Kristen Vondrak ◽

...

Keyword(s):

Type I ◽

Neuropathology in Mouse Models of Mucopolysaccharidosis Type I, IIIA and IIIB

PLoS ONE ◽

10.1371/journal.pone.0035787 ◽

2012 ◽

Vol 7 (4) ◽

pp. e35787 ◽

Cited By ~ 103

Author(s):

Fiona L. Wilkinson ◽

Rebecca J. Holley ◽

Kia J. Langford-Smith ◽

Soumya Badrinath ◽

Aiyin Liao ◽

...

Keyword(s):

Mouse Models ◽

Type I ◽

Anti-drug antibody response in mucopolysaccharidosis type I patients treated with AGT-181, a brain penetrating human insulin receptor antibody-iduronidase fusion protein

10.1016/j.ymgme.2017.12.343 ◽

2018 ◽

Vol 123 (2) ◽

pp. S126 ◽

Cited By ~ 3

Author(s):

Mathias Schmidt ◽

Ruben J. Boado ◽

Roberto Giugliani ◽

William M. Pardridge

Keyword(s):

Antibody Response ◽

Fusion Protein ◽

Insulin Receptor ◽

Human Insulin ◽

Type I ◽

Receptor Antibody ◽

Human Insulin Receptor

Evidence of a progressive motor dysfunction in Mucopolysaccharidosis type I mice

Behavioural Brain Research ◽

10.1016/j.bbr.2012.04.051 ◽

2012 ◽

Vol 233 (1) ◽

pp. 169-175 ◽

Cited By ~ 24

Author(s):

Guilherme Baldo ◽

Fabiana Quoos Mayer ◽

Barbara Martinelli ◽

Anna Dilda ◽

Fabiola Meyer ◽

...

Keyword(s):

Motor Dysfunction ◽

Type I ◽

69. Gene therapy of mucopolysaccharidosis type I: Intraventricular administration of adeno-associated virus vector transducing the human alpha-l-iduronidase gene in a murine model of the disease

10.1016/j.ymgme.2007.10.081 ◽

2008 ◽

Vol 93 (2) ◽

pp. 31

Author(s):

R. Scott McIvor ◽

Daniel A. Wolf ◽

Zhenhong Nan ◽

Mayra Quito ◽

Kelly Podetz-Pedersen ◽

...

Keyword(s):

Gene Therapy ◽

Murine Model ◽

Virus Vector ◽

Type I ◽

Intraventricular Administration ◽

Adeno Associated Virus

373. Expression of Human Iduronidase from Sleeping Beauty Engineered Human B Lymphocytes as a Cellular Therapy for Mucopolysaccharidosis Type I

Molecular Therapy ◽

10.1016/s1525-0016(16)33982-x ◽

2015 ◽

Vol 23 ◽

pp. S148

Author(s):

Eric J. Herbig ◽

Kendra Hyland ◽

Mei Xu ◽

Rian de Laat ◽

Erik Olson ◽

...

Keyword(s):

B Lymphocytes ◽

Cellular Therapy ◽

Sleeping Beauty ◽

Type I ◽

Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I

Proceedings of the National Academy of Sciences ◽

10.1073/pnas.1413645111 ◽

2014 ◽

Vol 111 (41) ◽

pp. 14894-14899 ◽

Cited By ~ 24

Author(s):

C. Hinderer ◽

P. Bell ◽

B. L. Gurda ◽

Q. Wang ◽

J.-P. Louboutin ◽

...

Keyword(s):

Gene Therapy ◽

Type I ◽