Following discussions with regulatory authorities in the U.S. and Europe, Idorsia has outlined a new Phase 3 registration program…
Articles by Marisa Wexler, MS
People with Fabry disease have abnormalities in the blood vessels within the eye, and these abnormalities may be indicative…
The experimental gene therapy AMT-191 appears to be working as intended in an early clinical trial, developer uniQure said,…
Sangamo Therapeutics has begun seeking accelerated approval from the U.S. Food and Drug Administration (FDA) for isaralgagene…
Markers of disease-related tissue damage in the brains of Fabry disease patients treated with…
Health Canada has approved the enzyme replacement therapy Elfabrio (pegunigalsidase alfa) to treat adults with Fabry disease. “With…
Chiesi Global Rare Diseases and Protalix Biotherapeutics are asking a committee of the European Medicines Agency (EMA) to…
Enzyme replacement therapy (ERT) may help limit damage to blood vessels in the kidneys of people with Fabry…
Treatment with the experimental gene therapy AMT-191 led to increases in levels of the enzyme whose deficit causes…
Looking at blood vessels in the eye in people with Fabry disease may give clinicians new insights into problems…