Clinician Bias Linked to Opioid Stigma in Sickle Cell Care
Negative descriptors in clinician notes for sickle cell patients are closely associated with stigma surrounding opioid use.
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Clinician Bias Linked to Opioid Stigma in Sickle Cell Care
Negative descriptors in clinician notes for sickle cell patients are closely associated with stigma surrounding opioid use.
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Positive Casgevy Data for Sickle Cell Disease in Younger Children
New clinical data for Casgevy in younger SCD/TDT patients age 5-11 show efficacy and safety consistent with older populations.
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Expanding Access to Million-dollar Sickle Cell Gene Therapies for Medicaid Patients
A federal program aims to expand Medicaid access to two FDA-approved gene therapies for sickle cell disease, which cost millions of dollars per patient.
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Sickle Cell Disease Study Tests Liquid Carbon Monoxide
A Phase 2a clinical trial will evaluate an oral low-dose liquid carbon monoxide (CO) therapeutic candidate for the treatment of sickle cell disease.
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New Genetic Target for Sickle Cell Disease Treatment
Researchers have identified a potential new gene target that could be edited to treat sickle cell disease.
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Novel Bone Marrow Transplant Can Cure Sickle Cell Disease
A novel transplant procedure using bone marrow from a “half-matched” donor is safe and curative for adults with sickle cell disease, as well as more affordable than recently approved gene therapy products for SCD.
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Sickle Cell Disease Tied to Older-Looking Brains
Researchers found that participants with sickle cell disease had brains that appeared an average of 14 years older than their actual age.
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Milder Blood Stem Cell Transplants Preserves Lung Function in Sickle Cell Patients
An NIH study found that lung function remained stable or improved after patients with sickle cell disease underwent low-intensity blood stem cell transplants.
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Cystic Fibrosis Foundation Funds Prime Medicine’s Gene Editing Research
The Cystic Fibrosis Foundation will provide up to $15 million to Prime Medicine for preclinical research into gene editing for cystic fibrosis.
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Five Pharma Approvals from 2023
In some of the most notable pharmaceutical approvals from 2023, drugs for RSV, CF, COVID-19, sickle cell disease, and opioid overdose received an FDA stamp in 2023.
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Multimillion Dollar Price Tags for Sickle Cell Gene Therapies
Two gene therapies approved by the FDA last week for the treatment of sickle cell disease will have multimillion dollar price tags for patients (and insurers), according to Reuters.
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FDA Approves First CRISPR-based Therapy for Sickle Cell Disease
The FDA approved the first cell-based gene therapies for the treatment of sickle cell disease in patients 12 years and older. One therapy, Casgevy, is the first FDA-approved treatment to utilize the genome editing technology CRISPR/Cas9
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Sickle Cell Eye Complications Affect Children at Same Rate as Adults
A study of kids with sickle cell disease revealed one-in-three had sickle retinopathy, suggesting children need to be screened for vision problems as often as adults.
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Severe Sickle Cell Patients Have High Risk Tolerance for Gene Therapies
Individuals living with severe sickle cell disease (SCD) are highly interested in new, potentially curative gene therapy treatments and are willing to accept associated risks for a chance at a cure, according to a study published in Blood Advances.
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Integrating Music Therapy Across a Large Health System
A study outlines how researchers at a large healthcare system integrated music therapy program, which has shown benefits for lung disease patients and pallative care.
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Lung Health: Lung Volume Abnormalities in Sickle Cell Patients
Young patients with sickle cell anemia demonstrated more abnormalities in lung volume via lung clearance index compared to healthy counterparts.
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Researching Pain in Sickle Cell Disease Patients
Researchers are studying how the autonomic nervous system—a network of cells that regulates processes we don’t consciously think about (like breathing and blood vessel constriction)—plays a role in sickle cell disease.
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Precise Pain Treatment for Sickle Cell Disease
Machine-learning strategies can be applied to routinely collected physiological data, such as heart rate and blood pressure, to provide clues about pain levels in people with sickle cell disease, according to new research.
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Alternatives to Opioids for Sickle Cell Disease Patients
An enriched diet and companionship can reduce pain in mice with sickle cell disease by increasing serotonin, researchers say.
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Sickle Cell Discovery May Provide Pathway to Treat COVID-19
Scientists have found a way to lower stroke risk in sickle cell patients that opens a gateway of potential treatments for inflammation and clotting stemming from conditions such as COVID-19.
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Oxbryta FDA-approved for Sickle Cell Disease
Sickle cell disease patients receiving Oxbryta (1500mg) experienced a 51.1% increase in hemoglobin response rate compared to 6.5% in the placebo group.
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FDA Approves New Treatment for Sickle Cell Disease
The FDA approved Endari (L-glutamine oral powder) for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder.
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Hypoxia, Nitric Oxide Worsen Sickle Cell Disease
Low levels of both oxygen and nitric oxide appear to have an unfortunate synergy for patients with sickle cell disease, researchers report.
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