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…

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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
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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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Could Base Editing Treat SCD?
Sourced from NIH Image Gallery (Public Domain). Credit: Ernesto del Aguila III, National Human Genome Research Institute, NIH

Could Base Editing Treat SCD?

Have you ever heard of base editing? This relatively new technology within the field of genome editing offers the potential opportunity to treat a variety of genetic conditions. According to…

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