In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare conditions. A "rare" condition is defined as one affecting fewer…
According to a story from Kalimpong News, over 300 children with rare diseases in India are still waiting for the release of pledged funds from the Union Health Ministry so…
Clinical trials are important in developing a better understanding of certain diseases or conditions, as well as how to treat them. According to HCP Live, researchers evaluated PRX-102 (pegunigalsidase alfa),…
Munique Slongo is a rare disease patient diagnosed with Fabry disease. She has taken the time to share her story about what life with this diagnosis has been like. Fabry…
Avrobio has just announced that they will be stopping work on their investigative gene therapy for Fabry disease based on unexpected and disappointing results from a Phase 2 clinical trial.…
From November 21st until the 23rd, medical professionals and others involved in the metabolic field will come together for the 14th International Congress of Inborn Errors of Metabolism (ICIEM). It…
Fabry disease patients may have another treatment option soon, according to a press release published in BioSpace. Protalix Biotherapeutics and Chiesi Global Rare Diseases have provided an update on the…
Written by Dawn Laney, MS, CDC, CCRC The journey to a Fabry disease diagnosis is rarely a clear, straight path. Fabry can mimic more common health issues and lead patients…
According to a story from BioPharma Dive, gene therapy for Fabry disease is still in its early stages of development. However, it is already beginning to show potential as a…
On August 2, 2021, biotechnology company Amicus Therapeutics ("Amicus") shared that its oral treatment Galafold (migalastat) was approved by the European Commission (EC) for the treatment of adolescent patients (ages…
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…
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