Fabry Disease News

June 10, 2022June 10, 2022

NORD’s ‘Living Rare, Living Stronger’ Patient, Family Forum Set for June 26

The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in ... Read more

June 3, 2022June 3, 2022

Galafold Safe, Effective at Reducing Heart Damage: Real-life Study

Fabry Disease News

Galafold (migalastat) is generally safe and significantly reduces disease-associated heart enlargement in Fabry disease patients carrying Galafold-amenable mutations, according to two-year data from a real-life, multicenter study in Germany. These ... Read more

May 27, 2022May 27, 2022

ECG Parameter May Help ID Fabry Patients at Risk for Pacemaker

Fabry Disease News

Many Fabry disease patients with heart involvement develop a slow and irregular heartbeat, called bradyarrhythmia, late in the disease course when heart damage is already advanced, according to a small ... Read more

May 20, 2022May 20, 2022

Fabry GLA Gene Variant Found in Parkinson’s Patients

Fabry Disease News

A variant in the GLA gene associated with Fabry disease was found in four women with Parkinson’s disease, but they had no Fabry symptoms, according to results from a study of 236 ... Read more

May 13, 2022May 13, 2022

Rare Autoimmune Kidney Disorder Seen With Fabry Disease

Fabry Disease News

The case of a man with Fabry disease that co-occurred with a rare autoimmune kidney disorder was described in a recent report. While it’s possible that the rare combination was ... Read more

May 6, 2022May 6, 2022

Organizations Rally to Help Ukrainian Rare Disease Patients

Fabry Disease News

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ... Read more

April 29, 2022April 29, 2022

PRX-102 Shows Safety, Efficacy Similar to Fabrazyme in Phase 3 Trial

Fabry Disease News

Two years of treatment with the experimental enzyme replacement therapy PRX-102 (pegunigalsidase alfa) prevented kidney decline in adults with Fabry disease and showed safety and effectiveness similar to Fabrazyme (agalsidase beta), ... Read more

April 22, 2022April 22, 2022

ERT Prevents Lung Health Decline With Classic Fabry, Study Suggests

Fabry Disease News

People with classic Fabry disease due to a common disease-causing mutation who don’t smoke can still have poorer lung health and symptoms like dry cough and shortness of breath, but these ... Read more

April 21, 2022April 21, 2022

Europe Awaits Proposed New Framework for Sharing Health Data

Fabry Disease News

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health ... Read more

April 15, 2022April 15, 2022

Telehealth Added to Latest Digital-friendly NORD State Report Card

COVID-19 News, Fabry Disease News

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues ... Read more

April 8, 2022April 8, 2022

Community Invited to Share Stories for Fabry Awareness Month

Fabry Disease News

Members of the Fabry disease community are invited to share their stories — those of patients, caregivers, family members, and advocates — and to use social media to celebrate Fabry ... Read more

April 1, 2022April 1, 2022

Fabrazyme Biosimilar Marketed in Japan Under New Agreement

Fabry Disease News

JCR Pharmaceuticals and Sumitomo Dainippon Pharma have teamed up to market and commercialize in Japan agalsidase beta BS, a biosimilar of Fabrazyme, an already approved treatment designed to replace a ... Read more

March 25, 2022March 25, 2022

Higher Dose of FLT190, Fabry Gene Therapy, To Be Tested Next

Fabry Disease News

The next patient to receive FLT190, an investigational gene therapy for Fabry disease, in the ongoing Phase 1/2 MARVEL-1 trial will be given a second, higher dose of therapy, accelerating an ... Read more

March 25, 2022March 25, 2022

Increased Education Crucial to Improving Rare Disease Care, Survey Finds

Fabry Disease News

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over ... Read more

March 18, 2022March 18, 2022

ST-920 Gene Therapy Continues to Show Safety, Efficacy in Trial

Fabry Disease News

Updated findings in an ongoing Phase 1/2 clinical trial of Sangamo Therapeutics‘ gene therapy ST-920 (isaralgagene civaparvovec) shows the treatment continues to be well tolerated and effective in adults with Fabry ... Read more