Posts by: Alice Melao

News

Globotriaosylsphingosine (LysoGb3) blood levels can be a useful and accurate biomarker to diagnose and monitor Fabry disease, rather than only using GLA mutational status, researchers suggest. The study, “Mutations ... Read more

October 16, 2018October 16, 2018

Antioxidants May Help Prevent Damage to Heart, Kidneys in Fabry Patients, Research Suggests

News

Inhibition of oxidative compounds using pharmacological or nutritional approaches, in addition to enzyme replacement therapy, may benefit patients with Fabry disease, research suggests. Treatment with antioxidant agents may help ... Read more

July 31, 2018July 31, 2018

Chiesi Acquires Global Development, Sales Rights to PRX-102 as Treatment for Fabry Disease

News

Chiesi, an Italian pharmaceutical company, has acquired the exclusive rights to develop and commercialize PRX-102 (pegunigalsidase alfa) in the U.S. as an investigative therapy for Fabry disease. The agreement expands ... Read more

June 21, 2018June 21, 2018

MicroRNA Molecules in Blood May Serve as Biomarkers for Fabry Disease, Study Suggests

News

A specific set of small RNA molecules in the blood can help diagnose Fabry disease and monitor response to therapy, according to recent findings. The study, “A pilot study of ... Read more

June 19, 2018June 19, 2018

Nerve Cell Loss May Contribute to Sweating Problems in Fabry Patients, Especially Women

News

Reduced sweating observed in Fabry patients may be a consequence of nerve cell loss in sweat glands, particularly in women, a study has found. The study, “Quantification of sweat ... Read more

May 29, 2018May 29, 2018

Early, Multidisciplinary Treatment with ERTs Recommended to Improve Fabry Outcomes

News

Early therapeutic intervention with enzyme replacement therapies (ERTs), along with a multidisciplinary follow-up, should be initiated at a young age in Fabry patients to promote the best outcomes and ... Read more

May 24, 2018May 24, 2018

First Patient Enrolled in Idorsia’s Phase 3 Lucerastat Trial; More Sought

News

Idorsia has enrolled the first patient in its MODIFY clinical trial, which will evaluate the potential of investigative oral monotherapy lucerastat as a treatment for adult patients with genetically ... Read more

May 22, 2018May 22, 2018

Long-term ERT May Help Prevent Heart Problems in Japanese Fabry Patients, Study Finds

News

Early treatment with enzyme replacement therapies targeting the underlying cause of Fabry disease may help delay cardiac manifestations and heart disease progression in Fabry patients, according to Japanese researchers. ... Read more

May 17, 2018May 17, 2018

Early Treatment Initiation May Prevent Lung Function Decline in Fabry Disease Patients, Study Suggests

News

Increased levels of fat molecules characteristic of Fabry disease are linked to lung function decline over time. Progressive airflow limitation in Fabry patients can be prevented by starting treatment ... Read more

May 10, 2018May 10, 2018

One-third of Fabry Patients Experience Cognitive Impairments, Danish Study Finds

News

Approximately one-third of Fabry disease patients have cognitive impairments, a feature more common in those who have a deficient blood supply to the brain (called cerebrovascular disease), according to ... Read more