Editor’s Choice: A New Dystonia Center of Excellence, A Call for Sickle Cell Blood Donors, and a New Treatment Target for Glioblastoma

Happy Friday! A tropical storm system will be traveling up through the mid-Atlantic this weekend, bringing wind and rain. Hunker down! This week, we have stories on Duke University being…

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Two Novel Sickle Cell Disease Treatments Have Received Orphan Drug and Rare Pediatric Disease Designation

Global Blood Therapeutics (GBT) has just announced that their therapeutics called GBT021601 (GBT601) and inclacumab for sickle cell disease (SCD) have received both Rare Pediatric Disease Designation and Orphan Drug…

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Can Music Therapy Relieve SCD Pain?
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Can Music Therapy Relieve SCD Pain?

Many patients with sickle cell disease (SCD) experience “pain crises,” or periodic episodes of extreme pain. These pain crises occur when sickle-shaped red blood cells block the flow of blood…

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CRISPR Continues to Cut Impressive Numbers in Beta-Thalassemia, Sickle Cell Disease, and More

A year ago, Paddy Doherty’s doctor told him that he had a rare hereditary disease called transthyretin (ATTR) amyloidosis, the same disease that had killed his father. As reported in…

Continue Reading CRISPR Continues to Cut Impressive Numbers in Beta-Thalassemia, Sickle Cell Disease, and More
Patient Worthy’s One-Year-Checkup on Victoria Gray, the First Sickle Cell Disease Patient to Receive CRISPR in the US
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Patient Worthy’s One-Year-Checkup on Victoria Gray, the First Sickle Cell Disease Patient to Receive CRISPR in the US

   CRISPR is a gene-editing technique that has revolutionized the medical world with its approach to making precise changes in DNA. CRISPR found its match in 35-year-old Victoria Gray of…

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Crizanlizumab for SCD is Coming to England
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Crizanlizumab for SCD is Coming to England

According to The Guardian, England’s National Health Service (NHS) has announced that a new therapeutic option will be available for patients with sickle cell disease (SCD). This treatment, called crizanlizumab,…

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