Gene Therapy for Severe Beta Thalassemia Earns Conditional Marketing Approval in the EU
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Gene Therapy for Severe Beta Thalassemia Earns Conditional Marketing Approval in the EU

According to a story from BioPortfolio, the gene therapy company bluebird bio, Inc. recently announced that its gene therapy ZYNTEGLO™ has earned conditional marketing authorization in the European Union from…

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Orchard Therapeutics Acquires Global Licensing Agreement for Experimental Mucopolysaccharidosis Drug

According to a publication from Biospace, British clinical drug developer Orchard Therapeutics has secured global intellectual property rights to research, manufacture, and market a gene therapy program for the treatment…

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Bayer’s Aliqopa Receives Breakthrough Therapy Status for Marginal Zone Lymphoma
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Bayer’s Aliqopa Receives Breakthrough Therapy Status for Marginal Zone Lymphoma

According to a publication from Benzinga, the U.S. Food and Drug Administration has granted Breakthrough Therapy status to Bayer's relapsed follicular lymphoma drug Aliqopa (copanlisib) for the treatment of marginal…

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Canada’s Rare Disease Healthcare Policies Need to be Revised to Include Caregivers in the Discussion

The Problem One in every 12 Canadians will be diagnosed with a rare disease in their lifetime. Two/thirds of these individuals are children and most of these patients experience debilitating…

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Wheelchair Bound Spina Bifida Patient Finishes Top 12 in National CrossFit Competition After One Year of Training

Sydney Mccallister is a 32-year-old woman diagnosed with spina bifida. Essentially, the condition means that the neural tube has not closed all the way during the first month of embryonic…

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The Challenge: He is A Father, A Caregiver and A Scientist Trying to Save his Son and Others With ME/CFS or Chronic Fatigue Syndrome

  Ron Davis and his gene-sequencing technologies were mentioned years ago in The Atlantic alongside Elon Musk (SpaceX) and Jeff Bezos of Amazon fame. Since then, Davis has amassed over…

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