After 10 Children with Rare Diseases died, Indian MP Varnum Gandhi Warns that more Children are in Danger
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After 10 Children with Rare Diseases died, Indian MP Varnum Gandhi Warns that more Children are in Danger

  New Delhi: Recent headlines in the Hindustan Times highlighted a letter written by the leader of the Bharatiya Janata Party, Varnum Gandhi, to Health Minister Mansukh Mandaviya urging him…

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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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