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The Latest Breaking News from Sickle Cell Disease

Fulcrum Therapeutics Stock Rally Ignited By Promising Sickle Cell Results

Fulcrum Therapeutics Inc. (NASDAQ:FULC) stock is surging on Monday, after the company released initial results from the ongoing 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease.

Benzinga

Vertex's Gene Therapy Works In Children With Blood Disorders

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) on Saturday presented data from multiple studies demonstrating the clinical benefits of Casgevy (exagamglogene autotemcel) in patients aged 5 years and older with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).

Benzinga

Why Is Agios Pharmaceuticals Stock Sinking Today?

Agios Pharmaceuticals Inc. (NASDAQ:AGIO) stock plunged on Wednesday after the company released topline results from RISE UP Phase 3 data for mitapivat in sickle cell disease.

Benzinga

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Pfizer's Investigational Drug Flunks Late-Stage For Sickle Cell Disease

Pfizer Inc. (NYSE:PFE) on Friday released results from the Phase 3 THRIVE-131 study evaluating inclacumab, an investigational P-selectin inhibitor, in patients 16 years of age and older with sickle cell disease.

Benzinga

Student wins Hugh Cudlipp Student journalism award for work on sickle cell disease

The prize named after the late Mirror editor is dedicated to recognising outstanding journalistic work by editorial students

Evening Standard

UK sickle cell patients ‘get worse care than sufferers of similar disorders’

Study commissioned by NHS finds that sickle cell disease care lags behind that for conditions such as cystic fibrosis

The Guardian - UK

Sickle cell patients to have quicker and more accessible treatment in England

Government announces £9m funding to make specialist blood machines more widely available across NHS

The Guardian - UK

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Nottinghamshire families left unaware of babies’ blood test results in second NHS error

About 300 families may not have been told whether children carry trait for genetic disorders such as sickle cell disease

The Guardian - UK

Graham Serjeant obituary

Physician and researcher who dedicated his life to the study of sickle cell disease and its variants

The Guardian - UK

Dangerous 'crises' in sickle cell disease may be amplified by menstrual cycle

In a new study, researchers found that the inflammation behind painful episodes in sickle cell disease are correlated with the menstrual cycle.

LiveScience

Sickle cell disease is a genetic disorder that causes lifelong suffering – here’s what you need to know

Right now, approximately 20 billion red blood cells are busy travelling through your blood vessels. They are delivering oxygen to all the different tissues in your body and removing carbon dioxide to be breathed out of your lungs.

The Conversation

‘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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Benzinga

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.

The Independent UK

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

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The Guardian - UK

‘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 Hindu

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.

USA Today Sports Media Group

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.

Evening Standard

'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.

The Independent UK

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

Dynamite News

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:

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.

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