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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Diagnostic Timeline for Lysosomal Storage Disorders like Fabry Disease Significantly Reduced in South Africa

The Problem Lysosomal storage disorders (LSDs) affect approximately one out of every 7,000 people. There are around 50 different types of LSDs. These include Fabry disease, Batten disease, Gaucher disease,…

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Funding Goal Achieved for Pulmonary Arterial Hypertension Clinical Trial
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Funding Goal Achieved for Pulmonary Arterial Hypertension Clinical Trial

According to a story from Euroinvestor, the drug development company Resverlogix Corp. recently announced that it has received the necessary $2.9 million in funding for a Phase 2 clinical trial…

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The Argument for Mandatory Newborn Screening and Government Subsidies for Rare Diseases in India

The State of Rare Disease in India There are 7,000 known rare diseases worldwide. The Organization for Rare Diseases India (ORDI) estimates 70 million people are affected by a rare diagnosis…

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Experimental Fabry Disease Treatment Gains Orphan Drug Designation

According to a story from Fabry Disease News, the drug developer Avrobio recently announced that the US Food and Drug Administration (FDA) has given the company's experimental drug AVR-RD-01 Orphan…

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A Recap on 2018: Record Number of Drugs Approved by the FDA for Rare Diseases

Each year the Food and Drug Administration approves dozens of new therapies to fulfill unmet needs for various patient populations. It's just been published that in 2018 they approved a…

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This Just In: India Never Implemented their Rare Disease Policy as Promised
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This Just In: India Never Implemented their Rare Disease Policy as Promised

We all got really excited last year when India implemented a new plan to benefit rare disease patients. It was called the National Rare Disease Policy. Basically, it put 12.86…

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Critical Medication for Fabry Disease Gets Added to Australia’s Life Saving Drugs Program
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Critical Medication for Fabry Disease Gets Added to Australia’s Life Saving Drugs Program

According to a story from The Sydney Morning Herald, the Australian government recently announced that it will be adding Galafold, a drug used to treat rare Fabry disease, to the…

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The FDA is Modernising Their Drug Development Processes
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The FDA is Modernising Their Drug Development Processes

In a recent statement, Scott Gottlieb, the Commissioner of the US Food and Drug Administration, has re-affirmed the organisation’s commitment to modernising drug regulatory pathways. New changes are hoped to…

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FDA Commissioner Releases Statement on Targeted Therapies, New Efficiency Measures, and Modernizing Development
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FDA Commissioner Releases Statement on Targeted Therapies, New Efficiency Measures, and Modernizing Development

According to a story from Pharma Voice, Dr. Scott Gottlieb, the current commissioner of the US Food and Drug Administration (FDA) has recently released a statement in regards to efforts…

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Program Aims to Help Diagnose Certain Rare Diseases More Quickly
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Program Aims to Help Diagnose Certain Rare Diseases More Quickly

According to a story from PR Newswire, a new program called The Lantern Project is working to provide a free testing program that allow doctors to more quickly diagnose patients…

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New Updates on Patients Taking Part in Studies of a Gene Therapy for Fabry Disease Have Been Released
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New Updates on Patients Taking Part in Studies of a Gene Therapy for Fabry Disease Have Been Released

Avrobio has released updates on patients in two clinical trials of a potential gene therapy for Fabry disease. According to the announcement, two patients taking part in a Phase 1…

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This Dad Climbed a Literal Mountain for His Daughter with Pompe Disease

The Malaysia Lysosomal Diseases Association (MLDA) was formed in 2011 by the hands of eight families. Their goal was to raise awareness and funds for those diagnosed with lysosomal storage…

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So Why DOES Canada have Scarcer Access to Rare Disease Therapies?
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So Why DOES Canada have Scarcer Access to Rare Disease Therapies?

According to a story from CheckOrphan, Durhane Wong-Rieger, the CEO of the Canadian Organization for Rare Disorders, says that the Canadian health system has been letting down the roughly three…

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