New Potential Primary Biliary Cholangitis Treatment Granted Breakthrough Therapy Designation

Researchers at GENFIT are excited about promising results from studies investigating a new potential therapy for PBC and NASH, two rare liver diseases. Primary Biliary Cholangitis Primary biliary cholangitis (PBC)…

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Animal Model Study Using CRISPR Could Lead to Successful Treatment of Rare Lung Diseases

According to a story from EurekAlert!, a group of researchers affiliated with Penn Medicine and Children's Hospital of Philadelphia recently conducted an experiment in which the scientists used CRISPR gene…

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New Personal Kinetigraph (PKG) Allows Parkinson’s Patients in the UK to Connect with their Specialists

  Although guidelines for Parkinson’s patients suggest that they visit a specialist every six months, according to a report in Parkinson’s News Today, the waiting period may be up to…

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Latest Data Reinforces Effectiveness of a Potential Gene Therapy for Type 1 Spinal Muscular Atrophy
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Latest Data Reinforces Effectiveness of a Potential Gene Therapy for Type 1 Spinal Muscular Atrophy

According to a story from inpublic.globenewswire.com, the Novartis company AveXis has released interim data from an ongoing Phase 3 clinical trial of Zolgensma, a gene therapy drug that is being…

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Possible Treatment for Marginal Zone Lymphoma Earns Orphan Drug Designation
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Possible Treatment for Marginal Zone Lymphoma Earns Orphan Drug Designation

According to a story from globenewswire.com, the biopharmaceutical company TG Therapeutics, Inc. recently made an announcement declaring that the US Food and Drug Administration (FDA) had awarded the company Orphan…

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Myasthenia Gravis Patient Didn’t Let Diagnosis Stop Her from Running in the 2019 Boston Marathon

When you’re diagnosed with a rare condition it can change the entire course of your life, at least at the beginning. But those living with diseases like these are fighters.…

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