Activating a mitochondrial protein called TRAP1 can help to improve the functionality of lysosomes, the cellular structures that are defective…
Articles by Marisa Wexler, MS
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…
The case of a man with Fabry disease that co-occurred with a rare autoimmune kidney disorder was described in…
The European Commission is expected to propose a new governing framework for health data next month, called the European Health…
Note: This story was updated Jan. 17, 2022, to note that Avrobio is deprioritizing its Fabry program. …
A man living with Fabry disease for more than two decades was found to have a second co-occurring genetic…
Protalix BioTherapeutics has requested a type A meeting with the U.S. Food and Drug Administration (FDA) to discuss a…