Fabry Disease News

May 26, 2020May 26, 2020

Fewer Antibodies Against PRX-102 Than Fabrazyme Likely, Trial Data Suggest

Fabry Disease News

Fabry disease patients may naturally have higher levels of neutralizing antibodies against Fabrazyme (agalsidase beta) than against Protalix BioTherapeutics‘ investigational PRX-102 (pegunigalsidase alfa), BALANCE Phase 3 trial data suggest. As antibodies ... Read more

May 12, 2020May 12, 2020

FDA Gives Fabry Gene Therapy, FLT190, Orphan Drug Status

Fabry Disease News

The U.S. Food and Drug Administration (FDA) has designated FLT190, an investigational gene therapy for Fabry disease,  an orphan drug to support and speed its development and possible review, Freeline Therapeutics, its developer, announced. ... Read more

May 6, 2020May 6, 2020

I Always Have Something to Be Thankful For

Fabry Disease News

As of today, my family has spent seven weeks staying at home due to the coronavirus pandemic. We are seven weeks into home schooling (with one fantastic week off for ... Read more

April 28, 2020June 1, 2020

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

COVID-19 News, Fabry Disease News

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their ... Read more

April 28, 2020April 28, 2020

A143T Mutation in GLA Gene Likely Causes Late-onset Fabry Disease, Study Suggests

Fabry Disease News

A mutation called A143T in the alpha-galactosidase A (GLA) gene is likely a cause of type 2 Fabry disease that manifests predominantly as heart problems, a study suggests. The study, “Cardiomyopathy associated ... Read more

April 22, 2020April 22, 2020

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Fabry Disease News

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called ... Read more

April 22, 2020April 24, 2020

Dietary Considerations for People with Fabry Disease

Fabry Disease News

Fabry disease is a rare genetic disease characterized by problems in breaking down a type of fat molecule called globotriaosylceramide (Gb3 or GL-3). Patients have mutations in the GLA gene ... Read more

April 21, 2020April 21, 2020

Axovant, Invitae Offering Free Genetic Testing, Counseling for Lysosomal Storage Diseases

Fabry Disease News

Axovant Gene Therapies is collaborating with Invitae to offer free genetic testing in the United States and Canada to children suspected of having a lysosomal storage disease. The Axovant sponsorship ... Read more

April 15, 2020April 15, 2020

COVID-19 Didn’t Cancel Fabry Disease Awareness Month

Fabry Disease News

I have started and stopped and restarted this column so many times that I have lost count. My brain is fuzzy, my mind can’t seem to settle, and my beloved routines ... Read more

April 14, 2020April 14, 2020

Study: PRX-102 Is Safe, Slows Kidney Disease in Fabry Patients Previously Taking Replagal

Fabry Disease News

One-year treatment with Protalix BioTherapeutics‘ investigational enzyme replacement therapy PRX-102 (pegunigalsidase alfa) is safe, well-tolerated and appears to more effectively slow kidney disease progression in Fabry disease patients previously treated ... Read more

April 9, 2020April 10, 2020

Fabry Disease and Pregnancy

Fabry Disease News

Fabry disease is a rare genetic disorder caused by mutations in a gene called GLA. This gene provides cells with the instructions necessary to make the alpha-galactosidase A enzyme. Without ... Read more

April 7, 2020May 8, 2020

Avrobio Pauses Patient Dosing in AVR-RD-01 Gene Therapy Trials Due to COVID-19

COVID-19 News, Fabry Disease News

In response to the COVID-19 pandemic, Avrobio has temporarily paused enrollment and dosing of new patients in Phase 1 and 2 trials testing its investigational gene therapy AVR-RD-01 for the ... Read more

April 6, 2020May 10, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

COVID-19 News, Fabry Disease News

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. ... Read more

March 31, 2020May 15, 2020

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

COVID-19 News, Fabry Disease News

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The ... Read more

March 24, 2020March 24, 2020

FLT190 Granted Orphan Drug Status in Europe as Potential Gene Therapy for Fabry Disease

Fabry Disease News

The gene therapy candidate FLT190 — designed to increase blood levels of the enzyme alpha-GAL A, which is affected in Fabry disease — has been granted orphan drug designation by ... Read more