4D-310, a new gene therapy for Fabry disease that is being developed by 4D Molecular Therapeutics…
Articles by Marisa Wexler, MS
New clinical trial data support the effectiveness of AVR-RD-01, Avrobio‘s investigational gene therapy for Fabry disease.
A mutation called A143T in the alpha-galactosidase AÂ (GLA) gene is likely a cause of type 2Â Fabry disease that…
A new evaluation tool, the FAbry STabilization indEX (Fastex), can help detect disease progression in individuals with Fabry disease,…
Galafold (migalastat) is an effective treatment for certain Japanese people with Fabry disease, with no apparent…
Detectable differences in blood vessel architecture in the eyes of people with Fabry disease could prove useful in diagnosing the…
Kidney biopsies can be instrumental in diagnosing Fabry disease, particularly in females for whom symptoms can vary widely, a new…
Even with treatment, people with Fabry disease often experience pain to an extent that it affects their quality of life,…
Testing alpha-galactosidase A enzyme activity may be a more cost-effective screening method for diagnosing Fabry disease than genetic tests,…
The screening tool FabryScan — consisting of a physical assessment and a questionnaire — may be useful in diagnosing Fabry…