FAB-GT biopsy data: 100% reduction of kidney substrate at 12 months in first patient dosed with plato ® gene therapy platform Gb3 is a fatty substrate that accumulates in the cells of Fabry disease ...

The first Fabry disease patient treated with Avrobio’s plato gene therapy platform in a phase 2 trial has experienced the complete clearance of toxic kidney substrate. Avrobio designed the plato ...

In patients with Fabry disease, hematopoietic stem cells are collected from the patient and then transduced with lentiviral vector carrying a functional version of the α-galactosidase A (GLA) gene ...

When we caught up with Avrobio at the J.P. Morgan Healthcare Conference, the company was three months out from its last Fabry disease update. Despite signs of efficacy—and one patient being weaned off ...

PHILADELPHIA, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced initial preclinical data from its investigational adeno-associated viral (AAV) gene therapy program ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO) (the “Company”), a Phase 2 clinical-stage gene therapy company, today announced clinical trial updates from the ongoing ...

AL01211, a novel oral GCS inhibitor, shows favorable safety and robust biomarker reduction in Phase 2 trial in Fabry patients AceLink Therapeutics, a clinical-stage biopharmaceutical company ...

Robert J. Hopkin, MD, clinical geneticist, Cincinnati Children's Hospital Medical Center, discusses current and emerging therapeutic and technological advancements in the management of Fabry disease.

All-cause graft failure in kidney transplant recipients with Fabry disease was about 30%, and improved when enzyme replacement therapy became available. Patients with Fabry disease (FD) who undergo ...

The use of patient registries for a variety of diseases and treatments has increased in recent years. 6 The Agency for Healthcare Research and Quality (http ...

~ Supraphysiological expression of α-Gal A activity maintained for over a year in longest treated patient as of data cutoff date ~ ~ Stable Lyso-Gb3 levels maintained post-dosing, regardless of enzyme ...

Consistent with the 87% reduction in the first evaluable kidney biopsy, there is complete clearance of Gb3 inclusions in second evaluable kidney biopsy in Fabry disease Phase 2 trial New Gaucher ...