Fabry disease patients who share the same genetic mutation in the GLA gene may experience different symptoms, a…
Articles by Joana Carvalho, PhD
A study in a Nordic population found no apparent clinical benefit in screening patients with idiopathic small fiber neuropathy or…
Roughly half of the patients diagnosed with Fabry disease experience white matter lesions, which tend to develop earlier in…
Fabry disease patients struggle with physical exercise — measured by a cardiopulmonary exercise test — due to an impairment in…
Japanese researchers have used stem cells from Fabry patients to create an innovative laboratory model that mimics heart disease in…
Lucerastat, an investigative oral therapy currently in late-stage clinical development at Idorsia Pharmaceuticals, successfully reduced the accumulation of a…