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 the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000 in grants, totaling…

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 in Japan found.  The study, “…

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 need to start treatment early, its researchers said. The study, “Variable clinical features of patients with…

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 company hopes to initiate in mid-2022 with U.S. Food and Drug Administration (FDA) agreement, will test the gene…

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 to add an extra layer of protection against viruses as common as a cold or as dangerous as COVID. They…

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 now owns worldwide rights to develop, manufacture, and commercialize gene therapy candidates for Fabry disease and Pompe…

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 expected toward the end of this year. Those who complete the trial…

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 with Fabry disease. This decision was not related to any concerns with the safety or effectiveness of PRX-102 shown…

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 was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

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 enrolling men with Fabry at four sites across the U.S. These study locations are the Children’s…