Posts by: Alice Melao

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Analysis of dry blood drops may help diagnose Fabry disease and begin early treatment in patients who are experiencing cardiac, renal, or neurological symptoms of unknown cause, according to ... Read more

August 7, 2019August 7, 2019

Galafold Approved to Treat Fabry Teens and Adults in Argentina, Amicus Announces

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Adolescents and adults with Fabry disease who live in Argentina can now be treated with Amicus Therapeutics’ oral precision medicine Galafold (migalastat). The Agency of Medicines, Food and Medical ... Read more

July 24, 2019July 24, 2019

Gene Therapy AVR-RD-01 Effectively Prevents Buildup of Toxic Gb3 in Fabry Patients, Interim Data Show

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Avrobio’s investigational gene therapy AVR-RD-01 shows potential in promoting durable beneficial effects to treat Fabry disease, with enhanced therapeutic activity compared with the standard-of-care enzyme replacement therapy, according to ... Read more

June 19, 2019June 19, 2019

Protalix, Chiesi Preparing to Request Accelerated FDA Approval of PRX-102 for Fabry Disease

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Protalix BioTherapeutics and Chiesi Farmaceutici are planning to submit a biologics license application with the U.S. Food and Drug Administration (FDA) for PRX-102 (pegunigalsidase alfa) as a treatment for ... Read more

February 13, 2019February 13, 2019

Phase 3 Trial Shows Increased Stability of PRX-102 Over Other ERTs for Fabry Disease

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PRX-102 (pegunigalsidase alfa) is safe for the treatment of patients with Fabry disease and has better stability over other available therapies, according to interim data from a Phase 3 ... Read more

December 18, 2018December 18, 2018

LysoGb3 Blood Levels Can Help Diagnose Fabry Disease, Large Study Shows

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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

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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

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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

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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

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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

Posts by: Alice Melao

Alice Melao

Dried Blood Drops May Help Screen At-risk Patients Early for Fabry Disease, Study Suggests

Galafold Approved to Treat Fabry Teens and Adults in Argentina, Amicus Announces

Gene Therapy AVR-RD-01 Effectively Prevents Buildup of Toxic Gb3 in Fabry Patients, Interim Data Show

Protalix, Chiesi Preparing to Request Accelerated FDA Approval of PRX-102 for Fabry Disease

Phase 3 Trial Shows Increased Stability of PRX-102 Over Other ERTs for Fabry Disease

LysoGb3 Blood Levels Can Help Diagnose Fabry Disease, Large Study Shows

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

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

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

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

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