Dan Baker, a 56 year old former anesthesiologist, knew from childhood that something peculiar and unpleasant was going on with his body. Unfortunately, it would take decades before he found…
According to a story from Fabry Disease News, the month of April is recognized as Fabry Disease Awareness Month, a time dedicated to spreading awareness about Fabry disease in the…
It is estimated that one person in forty to sixty thousand is affected by Fabry disease. Approximately five hundred Canadians are known to have Fabry disease. Newswise recently carried the…
New research suggests that ultrasounds could be a new diagnostic tool for patients with a rare lysosomal storage disorder. According to Fabry Disease News, kidney dysfunction is characteristic in Fabry…
AVROBIO has released data from the ongoing Phase 2 FAB-GT trial, which is investigating AVR-RD-01 as a treatment for Fabry disease. This ex vivo lentiviral gene therapy was shown to…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Written by Kendra J. Bjoraker, Ph.D., L.P. Living well with a chronic condition such as Fabry can seem contradictory. Following diagnosis, you don’t have a shelf in your mind that…
Across the world, various countries have Orphan Drug or Orphan Medicinal Product designations that leading agencies grant to products designed to treat patients with rare diseases. Recently, AVROBIO announced that…
According to a press release from the American Kidney Fund (AKF), the organization, which is dedicated to fighting kidney disease, has partnered with the biotech company Sanofi Genzyme in a…
According to a story from PR Newswire, Chiesi Global Rare Diseases and the biopharmaceutical company Protalix BioTherapeutics, Inc. have recently announced that they have launched an Expanded Access Program (EAP)…
To determine the safety and efficacy of experimental gene therapy ST-920, researchers first examined the impact of the drug on mouse models of Fabry disease. As reported in Fabry Disease…
Protalix BioTherapeutics has recently completed the treatment portion of their Phase III BRIGHT trial, which is investigating PRX-102 as a treatment for Fabry disease. The data is still being collected…
According to a story from Fabry Disease News, any member of the Fabry disease patient community should strongly consider signing up for the Fabry Registry. Getting involved comes with several…
According to a story from Fabry Disease News, a recent case study analyzed the situation of two cousins living with Fabry disease who were being treated with enzyme replacement therapy,…
As reported in Business Fortnight, the top level gene therapy company AVROBIO has announced successful clinical trials for investigational drugs for Fabry disease and cystinosis. The CEO of AVROBIO, Geoff…
According to a story from prnewswire.com, the Chiesi Global Rare Diseases and the biopharmaceutical company ProTalix Biotherapeutics have recently announced that they have submitted a Biologics License Application (BLA) for…
Protalix BioTherapeutics has recently announced the results from the third phase of the BRIDGE trial, which evaluated pegunigalsidase alfa for the treatment of Fabry disease. As these results were positive,…
According to Batten Disease News, Invitae and Axovant Gene Therapies partnered up to offer free genetic testing in North America. This will focus on children who are thought to…
Fabry disease brings symptoms that can affect multiple organs throughout the body. The kidneys and gastrointestinal system are commonly affected, which can make life difficult. According to an article from…
According to Fabry Disease News, patients with Fabry disease may have better outcomes when treated with enzyme replacement therapy PRX-102 over Replagal. In a one year comparison, PRX-102 seems…
April is recognized as Fabry Disease Awareness Month by the Fabry community in the US and several other countries around the world. Advocacy groups such as the Fabry Support &…
As originally reported in Fabry Disease News, the potentially fatal genetic disorder Fabry disease can be diagnosed most effectively in infancy by newborn screening programs. By identifying the disease early,…
Sanofi There are an estimated 350 million rare disease patients across the world. Many conditions still don't have treatments. However, there are scientists dedicating their lives solely to this endeavor.…
According to a story from Financial Buzz, the gene therapy company AVROBIO recently presented updated data on February 10th, 2020 at the 16th Annual WORLDSymposium being held in Orlando, FL. These updates…
According to a story from RT, the Lobanov family of Nizhny Novgorod, Russia has been ravaged by Fabry disease. After a bureaucratic quagmire that has gone on for around three…
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