Fabry Disease

Latest News

December 27th 2021

Top 5 Most-Read Fabry Disease Articles of 2021

By Christina Mattina

December 12th 2021

Review Provides Overview of Clinical Picture, Diagnostic Confirmation of Fabry Disease

By Lauren Massaro

December 5th 2021

Case of 59-Year-Old Highlights Variability in Fabry Disease Presentation

By Jared Kaltwasser

November 29th 2021

Cerebral Small Vessel Disease and Stroke Risk in Fabry Patients

By Rose McNulty

November 27th 2021

CRISPR-Created Model of Fabry Disease Sparks New Insights

By Jared Kaltwasser

Video Interviews

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December 17th 2021

Dr Robert Hopkin on Future Considerations for Management of Fabry Disease

December 9th 2021

Dr Robert Hopkin Discusses Misconceptions in the Management of Fabry Disease

December 2nd 2021

Dr Robert Hopkin on Unmet Needs in Fabry Disease

November 26th 2021

Dr Robert Hopkin Addresses HRQOL Factors in Fabry Disease

November 19th 2021

Dr Robert Hopkin on Emerging Therapeutic, Technological Advancements in Fabry Disease

November 5th 2021

Dr Robert Hopkin Reviews Current Standards of Care in Fabry Disease

More News

November 11th 2021

Patients With Fabry Likely Experience Accelerated Atherosclerosis, Study Finds

Rose McNulty

A recent review highlighted the importance of managing cardiovascular risk factors in patients with Fabry disease versus the general population.

November 9th 2021

Study: Left Atrial Strain Could Be a Sign of Fabry Cardiomyopathy

Jared Kaltwasser

The parameter could potentially help clinicians zero in on the cause of left ventricular hypertrophy.

November 3rd 2021

Migalastat Preserved Renal Function Over Long Term in Patients With Fabry Disease

Jared Kaltwasser

New data show the therapy led to minimal changes in renal function, even after more than 8 years.

October 30th 2021

Patients With Fabry Disease Have a Poor QOL, Survey Finds

Laura Joszt, MA

Even compared with other patients with chronic diseases, patients with Fabry disease had a very poor quality of life.

October 29th 2021

Study Suggests Improved Screening Strategies for Fabry Disease

Allison Inserro

Early diagnosis of Fabry disease is essential and that since symptoms depend on the type of disease and sex and age of the patient, a high-risk screening system should account for the age of the target population, the researchers said.

October 16th 2021

Understanding HRQOL Trajectories in Patients With Fabry Disease

Laura Joszt, MA

Most patients with Fabry disease reported decreased physical and mental health-related quality of life (HRQOL) over 13 years of follow-up.

October 8th 2021

Review: Multidisciplinary Care Is Crucial to Fabry Disease Management

Rose McNulty

Pain is a hallmark of many Fabry disease cases, and improvements in management could dramatically improve quality of life for patients.

May 10th 2021

Agalsidase-β Improves Vascular Function, Blood Pressure in Fabry Disease

Jared Kaltwasser

A small study, based on 4 family members with Fabry disease, affirms the importance of early enzyme replacement therapy.

April 30th 2021

New Studies Affirm Safety Profile, Efficacy of Agalsidase Beta in Fabry Disease

Jared Kaltwasser

About 1 in 3 patients experienced adverse drug reactions in the 2 new safety studies, aligning with earlier reports.

Kidney transplants in patients with Fabry disease has a high success rate

April 28th 2021

Analysis: Kidney Transplantation in Fabry Disease Has High Success Rate

Jared Kaltwasser

All-cause graft failure in kidney transplant recipients with Fabry disease was about 30%, and improved when enzyme replacement therapy became available.

April 23rd 2021

Most Patients With Fabry Disease Face Mild or Moderate Symptoms Upon COVID-19 Infection

Jared Kaltwasser

Despite concerns that patients with Fabry disease might be at heightened risk of severe symptoms from SARS-CoV-2, early data find that most patients experience mild or moderate symptoms.

Researchers examine barriers to family genetic testing for Fabry disease

April 21st 2021

Overcoming Barriers to Genetic Testing Key to Identifying More Cases of Fabry Disease

Jaime Rosenberg

These national and regional barriers, which range from cost of screening to patient education, have hindered the widespread use of screening, say the researchers.

April 15th 2021

Omalizumab Enables Fabrazyme Desensitization After Anaphylaxis, Report Says

Maggie L. Shaw

This is a case study of a male patient with Fabry disease who presented with severe anaphylaxis following re-initiation of full-strength treatment with intravenous agalsidase beta (Fabrazyme).

January 26th 2019

PAN Foundation Announces New Fabry Disease Assistance Fund

The Patient Access Network (PAN) Foundation has opened a new patient assistance program for people living with Fabry disease/

February 4th 2018

Gene Therapy for Fabry Disease to Advance to Phase 2 Clinical Trials

Kelly Davio

Biotechnology company Avrobio has completed a $60 million Series B financing to advance multiple gene therapies, including AVR-RD-01, a proposed single-dose lentiviral gene therapy for Fabry disease (FD).

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