The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
Articles by Patricia Inácio, PhD
Long-term treatment with Fabrazyme (agalsidase beta), an enzyme replacement therapy for people with Fabry disease, slows the progression of kidney…
A first patient has been treated with PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy for Fabry disease that is under…
Pain, negative health perceptions, and certain coping styles for dealing with daily stressors are all linked to a greater incidence…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…
PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy (ERT) for Fabry disease, is closer to accelerated approval after a…
A novel gene therapy developed by 4D Molecular Therapeutics can increase gene delivery to the heart without triggering an…
A single injection with candidate gene therapy FLT190 showed positive results in a mouse model of Fabry disease…
Fabry disease shares some manifestations with rheumatic diseases, which has raised concerns of wrongful diagnosis and consequent delayed treatment. Still,…
The U.S. Food and Drug Administration (FDA) has approved Avrobio’s clinical program for AVR-RD-01 as a gene therapy candidate…