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sickle cell disease

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The National Institutes of Health (NIH) Gateway Center in Bethesda, Maryland

Health Jul 12

Trump administration’s NIH funding cuts threaten research on sickle cell disease

By Ali Rogin, Kaisha Young

Health Dec 09

FILE PHOTO: Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. Photo by Andrew Kelly/Reuters
Watch 5:08
Why the FDA’s approval of revolutionary sickle cell gene therapy is a ‘big deal’

Sickle cell disease is a chronic, debilitating condition that affects nearly 100,000 Americans, most of them with African ancestry. Now, the FDA has approved a groundbreaking treatment for it that uses the gene-editing tool CRISPR. John Yang speaks with Yale…

By John Yang, Claire Mufson, Harry Zahn

Health Dec 08

Sickle cell disease is complex on its own, but black men with the illness battle its stigmas and stereotypes too
Gene therapies for sickle cell disease approved in U.S.

Regulators on Friday approved two new gene therapies for sickle cell disease that doctors hope can cure the painful, inherited blood disorder that afflicts mostly Black people in the United States.

By Laura Ungar, Associated Press

Health Nov 16

Sickle Cell Disease, Human Blood Smear with Sickled cells, 500X. This blood smear shows numerous bizarre, abnormally shaped, and sickled erythrocytes (red blood cells). This single DNA base change causes an abnormal hemoglobin which causes the erythrocytes to bend into a sickle shape that can block circulation in small blood vessels.
The world’s first gene therapy for sickle cell disease has been approved in the UK

In a statement on Thursday, the Medicines and Healthcare Regulatory Agency said it had approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020.

By Maria Cheng, Associated Press

Health Oct 31

The outside of the Food and Drug Administration headquarters is seen in White Oak, Md., on Monday, November 9, 2015. The FDA is a federal agency of the United States Department of Health and Human Services and has been in commission since 1906. Photo By Al Drago/CQ Roll Call
WATCH: Sickle cell gene therapy gets review from FDA advisory committee

If approved, it would be the first gene therapy on the U.S. market based on CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020.

By Laura Ungar, Associated Press

Nov 20

First CRISPR treatment for sickle cell, other blood disease shows early benefits in two patients

By Sharon Begley, Adam Feuerstein, STAT

The first two patients to receive a CRISPR-based treatment for the inherited blood disorders sickle cell disease and beta thalassemia have benefited from the experimental therapy and experienced only temporary and treatable side effects.

Continue reading

Nov 15

U.S. approves new drug to manage sickle cell disease, costs $85,000 to $113,000

By Linda A. Johnson, Associated PRess

The monthly infusion, which halves occurrences of sickle cell pain episodes, will carry a list price of roughly $85,000 to $113,000 per year, depending on dosing. Insured patients generally will pay less.

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Dec 30

Sickle cell patients, families and doctors face a ‘fight for everything’

By Jenny Gold, Kaiser Health News

Premature death, a dearth of treatments, mistreatment in emergency rooms and a woeful lack of funding are just a few of the problems.

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Jun 22

Prodigy’s death shines light on slow progress against sickle cell disease

By Sharon Begley, STAT

The death of the rap artist Prodigy (Albert Johnson) at only 42 this week, after a lifetime of suffering from sickle cell disease, was a reminder of the devastating cost of the sometimes fatal genetic disorder — and of the…

Continue reading

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