Early ERT Improves Pompe Disease Outcomes
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Early ERT Improves Pompe Disease Outcomes

Enzyme replacement therapy (ERT) is an approved treatment strategy for those with Pompe disease. It involves providing patients with alpha-glucosidase, which helps to break down glycogen. In addition to reducing…

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Researchers May Have Found a More Effective Pompe Disease Treatment
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Researchers May Have Found a More Effective Pompe Disease Treatment

According to News Medical, researchers from the University of Cincinnati presented findings on a potentially new and effective treatment option for Pompe disease at the virtual American Academy of Neurology…

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Data for an Experimental Pompe Disease Therapy to be Presented at Upcoming Conference
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Data for an Experimental Pompe Disease Therapy to be Presented at Upcoming Conference

According to a press release from Sanofi, platform and poster presentations focused on the company's investigational product candidate avalglucosidase alfa are slated to be made public at the 17th annual…

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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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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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AskFirst: A New Collaborative Program to Support Rare Disease Patients in Gene Therapy Clinical Trials
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AskFirst: A New Collaborative Program to Support Rare Disease Patients in Gene Therapy Clinical Trials

AskBio Asklepios BioPharmaceuticals (AskBio) was first founded in 2001. It is based out of North Carolina.  This company is dedicated to the research of gene therapies as potential therapeutic options…

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The Muscular Dystrophy Association Steps up for Rare Neuromuscular Diseases
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The Muscular Dystrophy Association Steps up for Rare Neuromuscular Diseases

According to a story from Charcot-Marie-Tooth News, the Muscular Dystrophy Association (MDA) may at first seem like a group that is solely focused on helping muscular dystrophy patients, but its…

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