Fabry Disease News

October 20, 2020October 20, 2020

Protalix, Chiesi Launch Expanded Access Program for PRX-102 in the US

Fabry Disease News

Protalix BioTherapeutics and Chiesi Global Rare Diseases have launched an expanded access program (EAP) in the U.S. for PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy (ERT) to treat adults ... Read more

October 13, 2020October 13, 2020

Kidney Foundation Opens Effort to Raise Awareness of Fabry Disease

Fabry Disease News

The American Kidney Fund (AKF) has opened a Fabry disease education and awareness campaign that encourages people with chronic kidney disease (CKD) who don’t know the underlying cause of their disorder ... Read more

September 29, 2020September 29, 2020

Using Telemedicine Ensures Patient Monitoring and Safety in Emergencies, Study Says

COVID-19 News, Fabry Disease News

In times of emergency, telemedicine, home-based treatments, and lab tests should be adopted whenever possible to help people with Fabry disease manage their condition while ensuring their safety, an observational ... Read more

September 15, 2020September 15, 2020

Long-Term Galafold Safe, Effective as Fabry Treatment, ATTRACT Extension Shows

Fabry Disease News

The results from a one-year extension study of the Phase 3 ATTRACT trial demonstrate the long-term safety and effectiveness of Galafold (migalastat) in people with Fabry disease with specific mutations. Importantly, ... Read more

September 9, 2020September 9, 2020

Activities Underway for Newborn Screening Awareness Month

Fabry Disease News

As it does each September during Newborn Screening Awareness Month, Baby’s First Test is sharing information and stories that highlight efforts throughout the U.S. to bring attention to newborn testing. Baby’s ... Read more

September 8, 2020September 8, 2020

ST-920 Gene Therapy Found Safe, Effective in Mouse Model; Supports First-In-Human Trial

Fabry Disease News

A single administration of the experimental gene therapy ST-920 in a Fabry disease mouse model safely and effectively increased the levels of the alpha-GalA enzyme — which is lacking in ... Read more

September 1, 2020September 1, 2020

FDA Reviewing Application of PRX-102 as Fabry Treatment

Fabry Disease News

The U.S. Food and Drug Administration (FDA) has accepted for review an application from Chiesi Global Rare Diseases and Protalix BioTherapeutics that asks for approval of PRX-102 (pegunigalsidase alfa) as a treatment ... Read more

August 25, 2020September 26, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Fabry Disease News

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input ... Read more

August 25, 2020August 26, 2020

FDA Puts Fabry Gene Therapy 4D-310 on Fast Track

Fabry Disease News

4D-310, a new gene therapy for Fabry disease that is being developed by 4D Molecular Therapeutics (4DMT), has been granted fast track designation by the U.S. Food and Drug Administration (FDA). ... Read more

August 18, 2020August 18, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Fabry Disease News

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The ... Read more

August 11, 2020August 12, 2020

Pain, Negative Health Perceptions Linked to Patient Depression, Study Finds

Fabry Disease News

Pain, negative health perceptions, and certain coping styles for dealing with daily stressors are all linked to a greater incidence of depression among people with Fabry disease, according to a ... Read more

August 10, 2020September 10, 2020

New Streaming Channel Showcases Rare Disease Films

COVID-19 News, Fabry Disease News

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films ... Read more

August 4, 2020August 4, 2020

New Mutation That Causes Fabry Found in GLA Gene

Fabry Disease News

Investigators have identified a new mutation in the GLA gene that caused Fabry disease in a 25-year-old man with end-stage kidney failure. His case was described in the study “Rare ... Read more

July 21, 2020July 21, 2020

Certain Fabry Patients May Switch from ERT to Galafold, Real-life Study Suggests

Fabry Disease News

Switching from enzyme replacement therapy (ERT) to Galafold (migalastat) is a valid and safe therapeutic option for Fabry disease patients with Galafold-amenable mutations, according to a real-life study from Italy. Notably, the ... Read more

July 14, 2020July 14, 2020

Alternative Therapies Needed for Patients Who Respond Poorly to ERT

Fabry Disease News

A case study of two cousins with Fabry disease who started producing high levels of neutralizing antibodies after enzyme replacement therapy (ERT) highlight the need for alternative therapies. The study, ... Read more