American Kidney Fund and Sanofi Genzyme Partner up for New Fabry Disease Campaign
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American Kidney Fund and Sanofi Genzyme Partner up for New Fabry Disease Campaign

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…

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Experimental Treatment for Fabry Disease to be Made Available Under Expanded Access
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Experimental Treatment for Fabry Disease to be Made Available Under Expanded Access

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)…

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Early Success in Trials for Experimental Gene Therapies for Fabry Disease and Cystinosis
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Early Success in Trials for Experimental Gene Therapies for Fabry Disease and Cystinosis

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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Fabry Disease: A Biologics License Application for Pegunigalsidase Alfa has Been Submitted to the FDA
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Fabry Disease: A Biologics License Application for Pegunigalsidase Alfa has Been Submitted to the FDA

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…

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Free Genetic Testing for Lysosomal Storage Diseases Provided by Invitae, Axovant
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Free Genetic Testing for Lysosomal Storage Diseases Provided by Invitae, Axovant

  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…

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Enzyme Therapy PRX-102 May Slow Kidney Disease in Patients with Fabry Disease
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Enzyme Therapy PRX-102 May Slow Kidney Disease in Patients with Fabry Disease

  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…

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Newborn Screening Tests Help Catch Fabry Disease Before Irreversible Symptoms Begin
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Newborn Screening Tests Help Catch Fabry Disease Before Irreversible Symptoms Begin

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,…

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Sanofi is Making Strides in Lysosomal Storage Disease Research

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.…

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AVROBIO Provides Updates on its Fabry Disease Program and Cystinosis Program
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AVROBIO Provides Updates on its Fabry Disease Program and Cystinosis Program

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…

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“It Took 23 Years to Find out I Have Fabry Disease:” A Rare Patient Story From The Czech Republic
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“It Took 23 Years to Find out I Have Fabry Disease:” A Rare Patient Story From The Czech Republic

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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Amicus Therapeutics Continue to Develop Investigative Treatments for Fabry, Pompe, and Batten Diseases
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Amicus Therapeutics Continue to Develop Investigative Treatments for Fabry, Pompe, and Batten Diseases

According to a publication from Fabry Disease News, Amicus Therapeutics has as many as 14 investigational drugs currently in development for the treatment of rare diseases including Fabry, Pompe, and…

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