Fabry Disease News

June 18, 2021June 18, 2021

Get Ready for NORD’s Patient and Family Forum, Set for June 26-27

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the ... Read more

June 18, 2021June 18, 2021

Film Animator’s New Mission: Calling Attention to ‘Fabry Fog’

Fabry Disease News

Wes Burian had a dream job as an animated film surfacing supervisor, bringing to life lovable characters like Po from “Kung Fu Panda.” But Burian, now 48, has Fabry disease, ... Read more

June 11, 2021June 11, 2021

New Dosing Protocol May Shorten Fabrazyme Infusions

Fabry Disease News

A new infusion protocol that gradually reduces the amount of time needed for the delivery of the Fabry disease therapy Fabrazyme (algasidase beta) is safe and well-tolerated, a study reports. The ... Read more

June 9, 2021June 9, 2021

Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases

Fabry Disease News

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in ... Read more

June 4, 2021June 4, 2021

PRX-102 at Least as Effective as Fabrazyme, Interim Data Show

Fabry Disease News

One year of treatment with PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy (ERT), was at least as effective as Fabrazyme (agalsidase beta) at preventing kidney function decline in adults with Fabry ... Read more

June 3, 2021June 3, 2021

Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill

Fabry Disease News

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in ... Read more

May 28, 2021May 28, 2021

Fabrazyme Safe, Effective in Japanese Study

Fabry Disease News

Fabrazyme (agalsidase beta) demonstrated an acceptable safety profile and resulted in sustained reductions in globotriaosylceramide (GL-3) levels over seven years among patients with Fabry disease, a large real-world study from ... Read more

May 21, 2021May 21, 2021

New Study Explores Variability in ERT Outcomes

Fabry Disease News

Enzyme replacement therapy (ERT) may not effectively manage all aspects of Fabry disease in all patients, particularly when started at more advanced ages, a new study indicates. The findings highlight the ... Read more

May 14, 2021May 14, 2021

Avrobio Planning Pivotal Trial of AVR-RD-01 Gene Therapy for Fabry

Fabry Disease News

Avrobio is in the planning stages of a clinical trial to support a request for approval of AVR-RD-01 as a first-line gene therapy for people with Fabry disease. This registration trial, which the ... Read more

May 7, 2021May 10, 2021

Despite COVID Vaccinations, Caution Still Crucial for Transplant Recipients

COVID-19 News, Fabry Disease News

Long before COVID-19 changed the world, organ transplant recipients were wearing masks to shield themselves from airborne threats. Immunosuppressive medicines, which often are a lifelong after a transplant, require these people ... Read more

May 7, 2021May 7, 2021

CANbridge Acquires Rights to Gene Therapy Candidates

Fabry Disease News

CANbridge Pharmaceuticals has entered a strategic collaboration and license agreement to gain global rights to LogicBio Therapeutics’ gene therapy candidates for rare diseases with limited treatment options. Under the agreement, CANbridge ... Read more

April 30, 2021April 30, 2021

Lucerastat Phase 3 Trial Fully Enrolled, Results Likely Late in Year

Fabry Disease News

A Phase 3 trial evaluating lucerastat, an investigational substrate reduction therapy for Fabry disease, is fully enrolled and underway in 118 adult patients. Results from the MODIFY trial (NCT03425539) are ... Read more

April 28, 2021April 28, 2021

FDA Rejects Quick Approval of PRX-102, Plant-based ERT for Adults

Fabry Disease News

The U.S. Food and Drug Administration (FDA) has rejected Protalix BioTherapeutics and Chiesi Global Rare Diseases’ request for accelerated approval of PRX-102 (pegunigalsidase alfa) as the first plant-based, every-other-week enzyme replacement therapy (ERT) for adults ... Read more

April 23, 2021April 23, 2021

Quick Approval for COVID Vaccines a Concern, Rare Disease Patients Say

COVID-19 News, Fabry Disease News

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine ... Read more

April 16, 2021April 16, 2021

First Patient Dosed in Phase 1/2 Trial of 4DMT’s Gene Therapy 4D-310

Fabry Disease News

A Phase 1/2 clinical trial evaluating the investigational gene therapy 4D-310, developed by 4D Molecular Therapeutics (4DMT) for people with Fabry disease, has dosed its first patient. The trial (NCT04519749) is currently ... Read more