A specific set of small RNA molecules in the blood can help diagnose Fabry disease and monitor response to…
Articles by Alice Melao
Reduced sweating observed in Fabry patients may be a consequence of nerve cell loss in sweat glands, particularly in women,…
Early therapeutic intervention with enzyme replacement therapies (ERTs), along with a multidisciplinary follow-up, should be initiated at a young…
Idorsia has enrolled the first patient in its MODIFY clinical trial, which will evaluate the potential of investigative oral…
Early treatment with enzyme replacement therapies targeting the underlying cause of Fabry disease may help delay cardiac manifestations and…
Increased levels of fat molecules characteristic of Fabry disease are linked to lung function decline over time. Progressive airflow…
Approximately one-third of Fabry disease patients have cognitive impairments, a feature more common in those who have a deficient…
A specific genetic mutation of the GLA gene results in the development of atypical late-onset Fabry disease, a study found.
Timely diagnosis of Fabry disease can prevent irreversible structural changes in the heart, a study reports. In their research, “…
A small fat molecule that accumulates in the tissues of Fabry disease patients may be used as a biomarker…