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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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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India Turns to Enzyme Replacement Therapy as it Seeks to Address Rare Diseases
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India Turns to Enzyme Replacement Therapy as it Seeks to Address Rare Diseases

According to a story from health.economictimes.indiatimes.com, the country of India is currently beginning to take more steps in order to address the dire unmet medical need of its rare disease…

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Takeda Pharmaceutical Launches Trio of Enzyme Replacement Therapies in India to Treat Lysosomal Storage Disorders
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Takeda Pharmaceutical Launches Trio of Enzyme Replacement Therapies in India to Treat Lysosomal Storage Disorders

According to a press release from Takeda Pharmaceutical Company published by For Press Release, the Company has launched a collection of enzyme replacement therapies for use in the treatment of…

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Study Finds That Despite Sperm Abnormalities, Male Fertility is Not Affected in Fabry Disease
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Study Finds That Despite Sperm Abnormalities, Male Fertility is Not Affected in Fabry Disease

According to a story from Fabry Disease News, a recent study has found that despite changes to sperm cells, the fertility of men with Fabry disease is not affected. The…

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Avrobio’s Experimental Fabry Disease Gene Therapy Shows Promise in Early Trials
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Avrobio’s Experimental Fabry Disease Gene Therapy Shows Promise in Early Trials

According to a press release from the Massachusetts-based biotechnology company Avrobio, Inc., clinical tests of the Company's experimental Fabry disease gene therapy, AVR-RD-01, have yielded impressive preliminary results. Though the…

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Study Suggests Russian Healthcare Providers May Be Critically Uninformed About Fabry Disease
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Study Suggests Russian Healthcare Providers May Be Critically Uninformed About Fabry Disease

According to a publication from Fabry Disease News, Russian researchers recently screened over five-and-a-half thousand patients for Fabry disease while they continued with prescribed hemodialysis. The researchers' study, published in…

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Gene Therapy for 12 Rare Diseases will be Investigated Thanks to this Industry-Academia Partnership

Extended Collaboration  Amicus Therapeutics has just announced that they are expanding their collaboration with the University of Pennsylvania's Perelman School of Medicine for the next five years. This collaboration is…

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FDA Approves Investigational New Drug Application for Experimental Fabry Disease Therapy
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FDA Approves Investigational New Drug Application for Experimental Fabry Disease Therapy

According to a publication from Fabry Disease News, the U.S. Food and Drug Administration (FDA) has approved American biotechnology company Avrobio's Investigational New Drug (IND) application for experimental Fabry disease…

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