The Fabry disease burden is variable but significant among adolescents and caregivers, affecting their quality of life and mental…
Articles by Marta Figueiredo, PhD
Galafold (migalastat) is generally safe and significantly reduces disease-associated heart enlargement in Fabry disease patients carrying Galafold-amenable mutations,…
Many Fabry disease patients with heart involvement develop a slow and irregular heartbeat, called bradyarrhythmia, late in the…
People with classic Fabry disease due to a common disease-causing mutation who don’t smoke can still have poorer lung health…
Beginning enzyme replacement therapy (ERT) before age 16 is associated with reduced kidney and heart damage — relative to no…
Fabry disease patients who have had a kidney transplant and are on immunosuppressive therapies may show a weaker response…
A single dose of 4D-310, an investigational gene therapy from 4D Molecular Therapeutics (4DMT), appears generally safe and restored…
FLT190, Freeline Therapeutics’ experimental gene therapy, led to sustained increases in alpha-galactosidase A (Gal A), the faulty enzyme in …
A single dose of Avrobio’s investigational gene therapy AVR-RD-01 continues to show a favorable safety and tolerability profile for up to…
Avrobio’s ongoing FAB-GT Phase 2 clinical trial, testing its experimental gene therapy AVR-RD-01 in adolescents and adults with…