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The Latest Breaking News from Sickle Cell Disease

‘Groundbreaking’ sickle cell disease treatment approved for NHS use in England

Clinical trials find one-time gene therapy exa-cel offers ‘functional cure’ in 96.6% of patients

The Guardian - UK

£1.65m gene-editing therapy offers hope of cure for some blood disorder patients

The treatment uses gene-editing tool Crispr, which earned its inventors the Nobel Prize in chemistry in 2020.

The Independent UK

NHS error meant hundreds of parents did not know children’s sickle cell status

More than 800 African-Caribbean heritage families in Derbyshire were not sent results of tests on their babies

The Guardian - UK

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Editas Medicine Presents Data From RUBY Trial At ASH 2024, Showing Reni-Cel Achieves 96% VOE-Free Rate, Sustained Increases In Hemoglobin And Fetal Hemoglobin, And Clinically Meaningful Pain And Function Improvements In Severe Sickle Cell Patients; Safety Profile Consistent With Myeloablative Conditioning

Editas Medicine Reports Updated Clinical Data from the RUBY Trial of Reni-cel in Patients with Severe Sickle Cell Disease at the American Society of Hematology (ASH) Annual MeetingGlobe NewswirePoster presentation at ASH

Benzinga

Beam Therapeutics Unveils New Safety, Efficacy Data From Sickle Cell Disease Candidate, Stock Gains

Beam Therapeutics unveiled BEAM-101 trial data at ASH, showing improved outcomes in sickle cell patients and promising preclinical BEAM-103 results.

Benzinga

Black man with sickle cell disease died ‘naked and alone’ in cell after complaining of breathing difficulties

The 43-year-old was found unresponsive in a cell on the healthcare unit at HMP Pentonville on 17 May 2022

The Independent UK

Health chiefs launch review to tackle ‘poor care’ faced by many young sickle cell disease sufferers

It is estimated that around 300 babies are born in the UK with sickle cell disease each year

The Independent UK

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‘Life-changing’ drug for sickle cell disease to be offered by NHS in England

Voxelotor, which can help reduce need for blood transfusions, is administered as a daily tablet

The Guardian - UK

How subpar treatment options allow sickle cell disease to persist | Explained

Access to healthcare for sickle cell disease in India, including gene-editing technology, raises equity and access concerns.

The Hindu

Beam Rockets 26%, Hitting A Year High, On Its Efforts To Take On Crispr's New Gene-Editing Drug

Beam stock launched a year-high Tuesday as enthusiasm grows for its next-generation form of gene editing, a rival to CRISPR.

Investors Business Daily

Former Patriot Devin McCourty honored with Hero Among Us award during Celtics-Lakers game

The Boston Celtics handed former Patriot Devin McCourty a Hero Among Us Award for his work to fight Sickle Cell Disease during Celtics-Lakers game.

USA Today Sports Media Group

'My beautiful son died after failures in sickle cell treatment. The NHS must improve'

Tyrone Airey, 46, died after being given a fatal dose of morphine at Northwick Park hospital in March 2021. A coroner later ruled that 'neglect' contributed to his death. His mother Lorraine is fighting to ensure no NHS patient ever has to go through the same again.

Evening Standard

Landmark sickle cell drug axed by regulators

Exclusive: Crizanlizumab was hailed as first new drug to be introduced in over 20 years for people living with sickle cell disease

The Independent UK

Over 1 crore people screened under National Sickle Cell Anaemia Elimination Mission

More than 1 crore people have been screened for sickle cell disease under the National Sickle Cell Anaemia Elimination Mission, an official statement issued on Tuesday said. Read further on Dynamite News:

Dynamite News

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WEKU

Sickle cell patient's journey leads to landmark approval of gene-editing treatment

In early December, the Food and Drug Administration approved a gene-editing treatment for sickle cell disease, the first for any illness. One patient helped pave the way.

The Guardian - UK

The 10 biggest science stories of 2023 – chosen by scientists

From insight into our human ancestry and breakthroughs on the moon to a flourishing of AI and terrifying new developments in the climate, it was a year of scientific drama

The Hindu

People with blood disorders not eligible for reservation in jobs, says Centre

Centre informs Parliament that people suffering from blood disorders like thalassemia, haemophilia and sickle cell disease are not eligible for reservation in government jobs under the Persons with Disabilities category.

The Independent UK

Promising new gene therapies for sickle cell are out of reach in countries where they're needed most

New gene therapies promise a cure for sickle cell disease but experts say they are out of reach in places that need them the most

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The Guardian - UK

Strokes, terrible pain and early death: can Zambia help beat sickle cell by screening babies?

Early diagnosis and treatment could save lives from the disease, which kills 50% of affected children under five

The Hindu

Game-changer: On approval for gene therapy to treat sickle cell disease and beta thalassemia

Landmark decisions that mark the beginning of gene therapy using the CRISPR-Cas9 tool to treat diseases that could otherwise be cured only through bone marrow transplantation

The Hindu

US approves two gene therapies for sickle cell disease

The U.S. Food and Drug Administration on Friday approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough CRISPR gene editing technology.

The Street

FDA approves the country's first gene-editing treatment

The Food and Drug Administration approved the country's first gene-editing drug to treat sickle cell disease.

The Independent UK

Two gene therapies for sickle cell disease approved in US

U.S. regulators have approved two gene therapies for sickle cell disease

WEKU

FDA approves first gene-editing treatment for human illness

The Food and Drug Administration approved two genetic treatments for sickle cell disease, including one that uses gene-editing. The approvals offer hope for patients and signal a new medical era.

The Independent UK

Chronic shortage of sickle cell nurses putting patients at risk, damning report warns

Previously, The Independent revealed that only half of healthcare professionals feel they have sufficient tools to manage the long-term damage that sickle cell disease brings.

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