Amicus Therapeutics’ first oral drug for the rare condition Fabry disease has been approved by the FDA, giving patients an alternative to conventional injected enzyme replacement therapy.

Amicus Therapeutics announced that it will showcase two oral presentations and 20 posters related to its research on Fabry and Pompe diseases at the 21st Annual WORLDSymposium™ 2025 taking place ...

For example, AvroBio, now Tectonic Therapeutic, ceased its Fabry disease gene therapy program for AVR-RD-01 in 2022 after variable responses were seen in a Phase I/II trial. Nonetheless ...

In May 2023, Elfabrio was approved by the FDA and EMA for Fabry disease. This is a $2 billion market dominated by three products [1] and one where legacy offerings present several shortcomings.

Methods: Data from medical records of the 44 heterozygous females followed at Cedars-Sinai Medical Center were compiled and analyzed for symptoms of Fabry disease. Quality of life data were also ...

Also in this episode, several Upstate and WNC families share their experiences with rare diagnoses. The Jones family is hosting an event in March to raise awareness for Fabry disease. Watch Part 1 ...

The company's clinical-stage product candidates are ST-920, a gene therapy product candidate, which is in Phase 1/2 clinical study for the treatment of Fabry disease; TX200, a chimeric antigen ...

June 10, 2024 — Scientists have grown 'mini-guts' in the lab to help understand Crohn's disease, showing that 'switches' that modify DNA in gut cells play an ... Major Cause of Inflammatory ...

It is hoped this will enrich the FindZebra.com search engine to enhance earlier and more accurate diagnosis of Gaucher and Fabry diseases by developing specialised search tools. “That’s a nice ...

CEO Bradley Campbell highlighted a strong year for Amicus, with $528 million in total revenue for 2024, representing 32% growth year-over-year. Galafold revenue reached $458 million, growing 18%, ...

Feb. 27, 2025 — A new study suggests a nasal spray developed to target neuroinflammation could one day be an effective treatment for traumatic brain injury (TBI). By studying the effects of the ...