Phase 2 Fabry Disease Clinical Trial Has Been Stopped Due to Negative Outcomes
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Phase 2 Fabry Disease Clinical Trial Has Been Stopped Due to Negative Outcomes

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.…

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14th ICIEM Conferences: Data to be Presented on Fabry Disease and Gaucher Disease Trials
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14th ICIEM Conferences: Data to be Presented on Fabry Disease and Gaucher Disease Trials

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…

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China Accepts CAN103 IND for Gaucher Disease
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China Accepts CAN103 IND for Gaucher Disease

In a recent news release, rare disease-focused biopharmaceutical company CANbridge Pharmaceuticals Inc. ("CANbridge") shared that the National Medical Products Administration (NMPA) of China accepted an Investigational New Drug (IND) application…

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FDA Accepts Priority Review NDA for Potential Niemann-Pick Disease Type C Drug
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FDA Accepts Priority Review NDA for Potential Niemann-Pick Disease Type C Drug

According to a story from globenewswire.com, the US Food and Drug Administration (FDA) recently accepted the New Drug Application (NDA) for arimoclomol. This application is for the use of the…

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Rare Disease Advocacy Group has Innovative Idea to Raise Awareness Amidst Social Distancing
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Rare Disease Advocacy Group has Innovative Idea to Raise Awareness Amidst Social Distancing

Gaucher Disease Awareness Month takes place every October, but amidst this new global pandemic, planning has taken place early to figure out how to make the month as successful as…

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New Drug Application for Niemann-Pick Disease Type C Treatment Submitted Under Rolling Review
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New Drug Application for Niemann-Pick Disease Type C Treatment Submitted Under Rolling Review

According to a story from globenewswire.com, the biopharmaceutical company Orphazyme A/S has recently announced that it has submitted its New Drug Application (NDA) for its experimental treatment arimoclomol to the…

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Arimoclomol Receives the Fast Track Designation for the Treatment of ALS
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Arimoclomol Receives the Fast Track Designation for the Treatment of ALS

CytRx Corporation has recently received the Fast Track designation for their ALS treatment, arimoclomol. This status was also given for the treatment of Niemann-Pick disease and sporadic Inclusion Body Myositis…

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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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