The Developers of an Experimental Pompe Treatment Have Received Advice From Regulatory Authorities
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The Developers of an Experimental Pompe Treatment Have Received Advice From Regulatory Authorities

Amicus Therapeutics is developing a treatment for Pompe disease through its AT-GAA program. They have recently announced that they have met with regulators from the European Union and Germany who…

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North Carolina Has Added Three More Conditions to Their Newborn Screening Program
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North Carolina Has Added Three More Conditions to Their Newborn Screening Program

Lawmakers in the U.S. state of North Carolina have expanded the state’s newborn screening testing to include three more conditions. These are Pompe disease, Mucopolysaccharidosis type 1 (MPS 1), and…

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Patients Are Being Asked for Their Opinions on Treatments For Lysosomal Storage Diseases
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Patients Are Being Asked for Their Opinions on Treatments For Lysosomal Storage Diseases

In an effort to better understand patient experiences, researchers at the Massachusetts General Hospital are surveying people with lysosomal storage diseases about how they view their treatments, reports CheckOrphan. The…

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Report: Pharma Can Resume Testing Drug Efficacy for Neurological and Respiratory Conditions
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Report: Pharma Can Resume Testing Drug Efficacy for Neurological and Respiratory Conditions

RespireRx Pharmaceuticals announced last month it was cleared to resume its ampakine CX717 clinical trials to evaluate the drug’s efficacy in several central nervous system and respiratory disorders, including Pompe…

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