Possible Treatment for Paroxysmal Nocturnal Hemoglobinuria Recommended for Marketing Approval in the EU
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Possible Treatment for Paroxysmal Nocturnal Hemoglobinuria Recommended for Marketing Approval in the EU

According to a story from Market Screener, the biopharmaceutical company Alexion Pharmaceuticals has recently announced some encouraging news regarding the status for ravulizumab (marketed as ULTOMIRIS) in the EU. The…

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Innovative New Test Could Allow for Inflammatory Bowel Disease Patients to Receive More Personalized Treatment
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Innovative New Test Could Allow for Inflammatory Bowel Disease Patients to Receive More Personalized Treatment

According to a story from EurekAlert!, a team of scientists affiliated with the University of Cambridge have pioneered a new test that could herald a new development in the treatment…

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Evidence Suggests The Spinal Muscular Atrophy Gene Therapy Could Help all Patients Regardless of Severity

According to a story from BNN Bloomberg, data from three studies suggest that Zolgensma, a gene therapy for spinal muscular atrophy developed by Novartis, could be useful to all patients…

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As Gene Therapy Becomes a Reality, Spark Therapeutics Looks at Challenges That Still Remain
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As Gene Therapy Becomes a Reality, Spark Therapeutics Looks at Challenges That Still Remain

According to a story from BioPharma Dive, the advent of gene therapy development at Spark Therapeutics has been five years in the making. At this juncture, it has been nearly…

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First Ever Treatment Approved for Transthyretin Amyloid Cardiomyopathy Will Cost $225,000 per Year

The first ever drug in the United States for treating transthyretin amyloid cardiomyopathy (ATTR-CM) has finally been approved by the FDA. It's amazing news for this patient community. But, you…

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Clinical Trial for Experimental Centronuclear Myopathy Drug Given the Green Light

According to a story from BioSpace, the drug development company Dynacure recently released an announcement in which the company stated that its application for a Phase 1/2 clinical trial had…

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