Trusted Resources: Education
Scientific literature and patient education texts
Fetal Therapies and Trials for Lysosomal Storage Diseases: A Survey of Attitudes of Parents and Patients
source: Orphanet journal of rare diseases
year: 2022
authors: Schwab ME,Brown JEH,Lianoglou B,Jin C,Conroy PC,Gallagher RC,Harmatz P,MacKenzie TC
summary/abstract:Lysosomal storage diseases (LSDs) are inherited metabolic disorders that may lead to severe multi-organ disease. Current ERTs are limited by anti-drug antibodies, the blood-brain barrier, and early disease onset and progression before ERT is started. We have opened a phase I clinical trial of enzyme replacement therapy (ERT) for fetuses with LSDs (NCT04532047). We evaluated the attitudes of parents and patients with LSDs towards fetal clinical trials and therapies.
organization: Fetal Treatment Center, University of California, San Francisco, CA, USA.DOI: 10.1186/s13023-022-02178-z
read more
BookmarkRelated Content
-
MPS 1: Where Are We NowDr. Sandra Kingma from the Centre for Ra...
-
The First Mucopolysaccharidosis Type VII in a Taiwanese Girl: A Case Report and Review of the LiteratureThe present study included the first cas...
-
Open-Label Phase 1/2 Study of Vestronidase Alfa for Mucopolysaccharidosis VIIVestronidase alfa is an enzyme replaceme...
-
Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis Type IMusculoskeletal findings in MPS can prog...
-
Real-World Data Assessment of Safety of Home-Based and Hospital/Outpatient-Based laronidase Enzyme Replacement Thera...Enzyme replacement therapy (ERT) is avai...
-
Elizabeth F. Neufeld, PhDBorn in France, Elizabeth Neufeld immigr...
-
Enzyme Replacement Therapy With Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: An Integrated Analysis o...Enzyme replacement therapy (ERT) improve...
Powered by
