A charitable program can help people with lysosomal storage disorders, such as Fabry disease, who live in underserved communities worldwide…
Articles by Marisa Wexler, MS
With data now reaching up to 3.5 years, benefits are continuing to be reported with the use of AVR-RD-01, …
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
Note: This story was updated Sept. 9, 2020, to reflect that PRX-102 is licensed to Chiesi for all markets, including…
4D-310, a new gene therapy for Fabry disease that is being developed by 4D Molecular Therapeutics…
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…