One year after treatment with 4D-310, an experimental gene therapy for Fabry disease, measures of heart health have improved…
Articles by Marisa Wexler, MS
A program that screened nearly 500 men with implanted pacemakers for Fabry disease found that none of them had…
ISU303, an experimental enzyme replacement therapy for Fabry disease, showed a good safety profile and some promising signs of…
One-time treatment with ST-920 (isaralgagene civaparvovec), an experimental gene therapy being developed by Sangamo Therapeutics, continues to be generally…
Implementing nationwide screening programs for Fabry disease in people with a heart condition called hypertrophic cardiomyopathy (HCM) may help…
Activating a mitochondrial protein called TRAP1 can help to improve the functionality of lysosomes, the cellular structures that are defective…
AL01211, a once-daily oral therapy being developed by AceLink Therapeutics for Fabry disease, has been granted an orphan drug designation…
Doctors who treat Fabry disease tend to underappreciate the effect that symptoms like abnormal sweating and digestive complaints can…
Mendelics and Takeda are partnering for a new program that aims to use a genetic sequencing strategy to…
A tissue analysis technique called CARS microscopy could be useful for identifying heart involvement in Fabry disease, according to…