Posts by: Patricia Inacio, PhD

Patricia Inacio, PhD

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.

June 12, 2019June 12, 2019

Novel Gene Therapy Better at Targeting the Heart Shows Promise for Fabry Disease, Preclinical Data Suggest

News

A novel gene therapy developed by 4D Molecular Therapeutics can increase gene delivery to the heart without triggering an immune response, according to preclinical data. The gene therapy candidate ... Read more

June 5, 2019June 5, 2019

Patients Being Recruited in Europe to Test Gene Therapy Candidate FLT190 After Promising Preclinical Results

News

A single injection with candidate gene therapy FLT190 showed positive results in a mouse model of Fabry disease by significantly increasing blood levels of GLA — the enzyme affected ... Read more

May 22, 2019May 22, 2019

Fabry Disease Unlikely to be Misdiagnosed for RA in Clinical Practice, Study Suggests

News

Fabry disease shares some manifestations with rheumatic diseases, which has raised concerns of wrongful diagnosis and consequent delayed treatment. Still, patients with this rare genetic disease are unlikely to ... Read more

May 15, 2019May 15, 2019

FDA Approves Avrobio’s Gene Therapy Program for Fabry Disease

News

The U.S. Food and Drug Administration (FDA) has approved Avrobio’s clinical program for AVR-RD-01 as a gene therapy candidate for the treatment of Fabry disease. Supported by the FDA’s ... Read more

January 8, 2019January 8, 2019

Protalix BioTherapeutics Completes Enrollment for PRX-102 Phase 3 Trial

News

Protalix BioTherapeutics announced it has completed enrollment of its Phase 3 trial evaluating PRX-102 in Fabry disease patients previously treated with Shire’s Replagal (agalsidase alfa). The BRIDGE trial (NCT03018730) is an open-label switch-over study that will ... Read more

September 27, 2018September 27, 2018

Stroke Linked to Fabry Disease After Check on Patient’s Family History

News

A case study detailing a younger man admitted to a hospital for a stroke — later attributed to Fabry disease — highlights the importance of family history in diagnosing ... Read more

August 28, 2018August 28, 2018

New Diagnostic Kit Can Screen Newborns for Fabry Disease Using Single Dried Blood Sample

News

A new diagnostic tool that can screen for up to six lysosomal storage disorders, including Fabry disease, in newborns is now available in the United States. PerkinElmer, the kit’s ... Read more

June 28, 2018June 28, 2018

Diagnostic, Treatment Options for Fabry Disease Summarized in Review

News

A recent review has summarized the current diagnostic tools and available treatments for Fabry disease, highlighting the challenges and potential new avenues to improve patients’ diagnoses and clinical outcomes. ... Read more

April 26, 2018April 26, 2018

Plant-Derived Enzyme is Potential Source for Enzyme Replacement Therapy

News

An enzyme structurally similar to alpha-galactosidase A (GLA), present in a close relative of the tobacco plant, was able to rescue Fabry disease patients’ cells. These findings suggest that plant alpha-galactosidases ... Read more

March 29, 2018March 29, 2018

Galafold Approved in Japan to Treat Fabry Disease, Amicus Announces

News

Galafold (migalastat) capsules of 123 mg were approved in Japan to treat Fabry disease patients, ages 16 and older, with mutations amenable to the treatment, Amicus Therapeutics recently announced. This move ... Read more

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