#sickle cell disease

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from www.nytimes.com
2 weeks ago
Medicine

First Sickle Cell Gene Therapy Patient, 12, Leaves Hospital

Kendric Cromer received the first gene therapy for sickle cell disease, representing a significant breakthrough in treatment.

Sickle cell gene therapies roll out slowly

FDA has approved the first genetic treatments for sickle cell disease, offering new hope to patients like Olaide Adekanbi.

What Is Sickle Cell Disease?

Sickle cell disease is caused by a point mutation in the HBB gene that impacts hemoglobin function and structure.

Meet the Advocates and Researchers Revolutionizing Sickle Cell Care

Widespread newborn screening is essential to combat sickle cell disease effectively in Nigeria.

New Sickle Cell Treatments Reach Patients after Years of Effort

Sickle cell disease is complicated, affecting treatment despite a single genetic cause.
Research is multifaceted, targeting multiple levels to address the disease's complexity.

Doctors cured her sickle-cell disease. So why is she still in pain?

Genesis Jones underwent a stem cell transplant to cure her sickle-cell disease, but continues to face significant health challenges and isolation post-treatment.

First Sickle Cell Gene Therapy Patient, 12, Leaves Hospital

Kendric Cromer received the first gene therapy for sickle cell disease, representing a significant breakthrough in treatment.

Sickle cell gene therapies roll out slowly

FDA has approved the first genetic treatments for sickle cell disease, offering new hope to patients like Olaide Adekanbi.

What Is Sickle Cell Disease?

Sickle cell disease is caused by a point mutation in the HBB gene that impacts hemoglobin function and structure.

Meet the Advocates and Researchers Revolutionizing Sickle Cell Care

Widespread newborn screening is essential to combat sickle cell disease effectively in Nigeria.

New Sickle Cell Treatments Reach Patients after Years of Effort

Sickle cell disease is complicated, affecting treatment despite a single genetic cause.
Research is multifaceted, targeting multiple levels to address the disease's complexity.

Doctors cured her sickle-cell disease. So why is she still in pain?

Genesis Jones underwent a stem cell transplant to cure her sickle-cell disease, but continues to face significant health challenges and isolation post-treatment.
moresickle-cell-disease

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

Access to gene therapies for sickle cell disease is limited in developing countries like India and Africa.
High prices and complex requirements make gene therapies inaccessible to most patients in these regions.

New sickle cell treatment has roots at Children's Hospital of Philadelphia

The FDA has approved the first gene therapy treatments for sickle cell disease, a blood disorder that primarily affects people of color.
The gene therapy was tested at Children's Hospital of Philadelphia and has shown promising results in patients.

'It's Transformed My Life': FDA Approves First Gene-Editing Treatment for Illness | KQED

The CRISPR treatment for sickle cell disease involves editing a gene with CRISPR and infusing the modified cells back into patients.
The therapy, Casgevy, is designed to be a one-time treatment that alleviates symptoms for a lifetime.
#gene therapy

New Sickle Cell Therapies Will Be Out of Reach Where They Are Needed Most

FDA approves groundbreaking gene therapies for sickle cell disease
Access to these therapies remains a challenge for patients in countries where sickle cell is prevalent

The First Crispr Medicine Is Now Approved in the US

Casgevy uses Crispr technology to modify patients' cells and produce healthy hemoglobin.
Casgevy has shown promising results in a clinical trial, with 29 out of 31 patients being free of pain crises for at least a year.

World's first gene therapy for sickle cell and thalassemia approved in the U.K.

The UK has authorized the first gene therapy treatment for sickle cell disease, offering potential relief to thousands of patients.
Casgevy, made by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine licensed using the gene editing tool.
Gene therapy offers a potential cure for sickle cell disease and thalassemia, which have previously only been manageable through difficult treatments.

The First Crispr Medicine Just Got Approved

Sickle cell disease and beta-thalassemia are painful, lifelong conditions that can be fatal.
Casgevy, a gene therapy treatment, aims to restore working hemoglobin in the body.
Trials have shown that Casgevy can reduce severe pain crises and the need for blood transfusions in patients with these conditions.

New Sickle Cell Therapies Will Be Out of Reach Where They Are Needed Most

FDA approves groundbreaking gene therapies for sickle cell disease
Access to these therapies remains a challenge for patients in countries where sickle cell is prevalent

The First Crispr Medicine Is Now Approved in the US

Casgevy uses Crispr technology to modify patients' cells and produce healthy hemoglobin.
Casgevy has shown promising results in a clinical trial, with 29 out of 31 patients being free of pain crises for at least a year.

World's first gene therapy for sickle cell and thalassemia approved in the U.K.

The UK has authorized the first gene therapy treatment for sickle cell disease, offering potential relief to thousands of patients.
Casgevy, made by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine licensed using the gene editing tool.
Gene therapy offers a potential cure for sickle cell disease and thalassemia, which have previously only been manageable through difficult treatments.

The First Crispr Medicine Just Got Approved

Sickle cell disease and beta-thalassemia are painful, lifelong conditions that can be fatal.
Casgevy, a gene therapy treatment, aims to restore working hemoglobin in the body.
Trials have shown that Casgevy can reduce severe pain crises and the need for blood transfusions in patients with these conditions.
moregene therapy

FDA Approves First CRISPR Treatment in U.S.

The U.S. FDA has approved the first gene therapy treatment based on CRISPR technology.
The treatment, called exa-cel, is approved for sickle cell disease and may also be approved for beta thalassemia.

FDA approves first CRISPR gene editing treatment that may cure sickle cell disease

Regulators have approved two new gene therapies for sickle cell disease that could potentially cure the inherited blood disorder.
The treatments are the first to be approved based on CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020.

FDA approves CRISPR-based therapy for sickle cell disease

The FDA has approved the first therapy using CRISPR gene-editing technology to treat sickle cell disease.
The technology has the potential to be applied to other blood disorders, certain cancers, and infectious diseases.

Breakthrough Gene Treatments For Sickle Cell Disease Approved

Regulators have approved two new gene therapies for sickle cell disease, offering hope for a cure for the painful blood disorder
The treatments are designed for patients 12 and older with severe forms of the disease, and one of them is the first therapy based on CRISPR gene editing

FDA approves first gene-editing treatments for human illness

The FDA has approved the first gene-editing treatments for sickle cell disease, marking a milestone in the field of gene-editing.
The treatments have the potential to provide more targeted and effective treatments for rare diseases with limited current options.

F.D.A. Approves Sickle Cell Treatments, Including One That Uses CRISPR

The Food and Drug Administration has approved the first gene editing therapy and conventional gene therapy for sickle cell disease.
Obstacles to treatment include limited access to authorized medical centers, individualized procedures, and high costs.

Possible Therapy for Sickle Cell Won't Cure Its Racist Legacy - Non Profit News | Nonprofit Quarterly

The FDA is reviewing an experimental gene therapy treatment for sickle cell disease.
Racism has significantly affected the healthcare and outcomes of sickle cell disease patients.

The CRISPR Era Is Here

Gene-editing technology CRISPR has successfully treated a patient with sickle-cell disease, providing hope for future treatments.
The treatment resulted in the patient living virtually symptom-free, with 29 out of 30 eligible patients experiencing zero pain crises after treatment.
#gene editing

The U.K. approved its first CRISPR gene-editing drugand the U.S. is expected to follow

U.K. regulators have approved the first-ever CRISPR gene-editing drug for the treatment of sickle cell disease and beta thalassemia.
The therapy has shown success in clinical trials and is expected to reach the U.S. by the end of the year.
The gene editing treatment modifies stem cells to boost the production of functional hemoglobin, providing a fix at the DNA level.

UK authorizes first gene therapy for treating sickle cell disease

The UK's MHRA has approved the use of a gene-editing therapy for patients with sickle cell disease and beta thalassemia.
The therapy, called Casgevy, is the first-ever approved therapy that uses CRISPR-based gene editing technology.
The therapy has shown high efficacy in relieving severe pain associated with these blood disorders and could potentially replace the current standard of care.

The U.K. approved its first CRISPR gene-editing drugand the U.S. is expected to follow

U.K. regulators have approved the first-ever CRISPR gene-editing drug for the treatment of sickle cell disease and beta thalassemia.
The therapy has shown success in clinical trials and is expected to reach the U.S. by the end of the year.
The gene editing treatment modifies stem cells to boost the production of functional hemoglobin, providing a fix at the DNA level.

UK authorizes first gene therapy for treating sickle cell disease

The UK's MHRA has approved the use of a gene-editing therapy for patients with sickle cell disease and beta thalassemia.
The therapy, called Casgevy, is the first-ever approved therapy that uses CRISPR-based gene editing technology.
The therapy has shown high efficacy in relieving severe pain associated with these blood disorders and could potentially replace the current standard of care.
moregene editing

U.K. Is First to Approve a CRISPR-Based Therapy, Covering Two Blood Disorders

The UK has authorized a gene-editing medicine for treating sickle cell disease and beta thalassemia, marking the first approval worldwide for a therapy based on CRISPR technology.
The therapy, known as Casgevy, involves editing a gene in a patient's stem cells to produce high levels of fetal hemoglobin.
Casgevy offers a potentially curative treatment option for these inherited blood disorders, reducing the need for chronic therapies and blood transfusions.

Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval

Regulators in Britain have approved the first treatment derived from CRISPR, called Casgevy, for sickle-cell disease and beta thalassemia.
About 2,000 patients in Britain are expected to be eligible for Casgevy treatment.
The FDA is expected to approve Casgevy for sickle-cell patients in the US in early December.

U.K. Becomes First Country to Approve a CRISPR Disease Treatment

UK medicines regulator approves CRISPR gene editing therapy for sickle-cell disease and -thalassaemia.
Casgevy completely relieved 28 out of 29 participants of pain from sickle-cell disease for at least one year after treatment.
For at least one year after treatment with Casgevy, 93% of participants with a severe form of -thalassaemia did not need a red-blood-cell transfusion.

Casgevy: UK approves gene-editing drug for blood disorders

The UK has approved a gene therapy using the Crispr gene-editing tool to treat sickle cell disease and beta thalassemia, a first in the world.
The treatment involves removing stem cells from a patient's bone marrow and using Crispr to disable the faulty gene, allowing the body to produce functioning haemoglobin.
Trials have shown promising results, with the majority of patients experiencing relief from symptoms and no longer needing blood transfusions.

CRISPR Therapy Gets U.K. Approval, the First in the World

Britain's medicines regulator has approved the world's first gene therapy for sickle cell disease, offering hope to thousands of patients.
Casgevy is the first treatment licensed using the gene editing tool CRISPR, and it targets the problematic gene in a patient's bone marrow stem cells.
Both sickle cell disease and thalassemia are caused by genetic mutations that affect the production of hemoglobin in red blood cells.
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