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)…
Happy Friday! Sept. 13-19 is Balance Awareness Week. It's intended to bring attention to balance-related vestibular disorders such as Meniere’s disease and vestibular migraine. Looking for ways to help out?…
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.…
As originally reported in European Pharmaceutical Manufacturer Magazine, Chiesi, an Italian pharmaceutical company, has announced they will be launching a new division for rare disease research and therapy innovation. The…
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…
According to a story from Fabry Disease News, a recent study has found that certain cells tend to age more rapidly in patients with Fabry disease when compared to unaffected…
Bohuslav Skoupý had trouble with his kidneys. He even had a transplant. He also had heart problems. In the end, it turned out actually by chance that Fabry disease was…
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