Hereditary Angioedema Patients in the UK Get Early Access to New Treatment
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Hereditary Angioedema Patients in the UK Get Early Access to New Treatment

According to a story from GlobeNewswire, the company BioCryst Pharmaceuticals, Inc., recently announced that its drug berotralstat (marketed as Orladeyo) will be available in the UK under the Early Access…

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New Study Pioneers Method to Measure Myalgic Encephalomyelitis Severity
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New Study Pioneers Method to Measure Myalgic Encephalomyelitis Severity

A recent study published in the Journal of Translational Medicine describes an accurate and objective approach that aims to evaluate the severity of myalgic encephalomyelitis, also known as chronic fatigue syndrome. This…

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New Children’s Book for Kids with Craniofacial Differences is Published! “Simon and Patty Go To Camp”
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New Children’s Book for Kids with Craniofacial Differences is Published! “Simon and Patty Go To Camp”

Patricia Simon Patricia Simon was born with a cleft lip and palate. She is now a nurse, a public speaker, a patient advocate, and a writer. She has a great…

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Experimental Treatment for Sickle Cell Disease Earns Orphan Drug Designation in the EU
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Experimental Treatment for Sickle Cell Disease Earns Orphan Drug Designation in the EU

According to a story from Street Insider, the biopharmaceutical company Forma Therapeutics Holdings, Inc., recently announced that its investigational therapy candidate FT-4202 has earned Orphan Drug designation from the European…

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Companion Diagnostics FoundationOne CDx and VITRAKVI Used to Treat Solid Tumors
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Companion Diagnostics FoundationOne CDx and VITRAKVI Used to Treat Solid Tumors

In late October, molecular information company Foundation Medicine, Inc. ("Foundation") shared that its diagnostic tool FoundationOne CDx was approved as a companion diagnostic for VITRAKVI (larotrectinib). While FoundationOne CDx will…

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Baylor College Team Discovers the Origin of Alpha-1 Antitrypsin Deficiency in Infants
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Baylor College Team Discovers the Origin of Alpha-1 Antitrypsin Deficiency in Infants

  A recent article in Science News highlighted a discovery by Richard Seifes, M.D. of Baylor College of Medicine.  Dr. Seifes has discovered the origin of a liver disease that…

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Experimental Treatment for Acid Sphingomyelinase Deficiency Shows Potential in Trials
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Experimental Treatment for Acid Sphingomyelinase Deficiency Shows Potential in Trials

In a press release from Sanofi, a global biopharmaceutical company, the company's experimental therapy candidate olipudase alfa has demonstrated the ability to substantially improve spleen volume and lung function in…

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Study Suggests Charcot Marie Tooth Disease Causes Structural Reorganization in the Brain
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Study Suggests Charcot Marie Tooth Disease Causes Structural Reorganization in the Brain

A recent study in Frontiers in Neurology published findings on brain changes in patients with Charcot Marie Tooth disease Type 1A, a rare neurological disease that affects the muscles. The researchers have…

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