#sickle-cell-disease

[ follow ]
#crispr

CRISPR genome-editing grows up: advanced therapies head for the clinic

Gene-editing therapies like Casgevy show long-lasting benefits for blood disorders but face challenges in accessibility due to complexity and cost.

The First Crispr Treatment Is Making Its Way to Patients

The first CRISPR-based treatment, Casgevy, has been approved for sickle cell disease and beta thalassemia, marking a significant advance in gene-editing therapies.

CRISPR genome-editing grows up: advanced therapies head for the clinic

Gene-editing therapies like Casgevy show long-lasting benefits for blood disorders but face challenges in accessibility due to complexity and cost.

The First Crispr Treatment Is Making Its Way to Patients

The first CRISPR-based treatment, Casgevy, has been approved for sickle cell disease and beta thalassemia, marking a significant advance in gene-editing therapies.
morecrispr

Black man with sickle cell disease died naked and alone' in cell

Quality journalism is vital for addressing urgent social issues, including prison healthcare and reproductive rights, and should be accessible to all.
#gene-therapy

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.

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.

First Day of a New Life' for a Boy With Sickle Cell

Kendric Cromer starts a hopeful journey free from sickle cell disease thanks to new gene therapy.

Gene Therapies Could Transform Treatment of Rare Blood Disorders - News Center

Gene therapy shows promising results in treating rare genetic blood disorders like beta thalassemia and sickle cell disease.

New Jersey college student suffering from sickle cell disease has newfound hope with gene therapy at CHOP

Gene therapy at Children's Hospital of Philadelphia offers new hope for sickle cell disease, potentially being a life-changing treatment.

First Patient Begins Newly Approved Sickle Cell Gene Therapy

Commercially approved gene therapy offers hope to sickle cell patients like Kendric Cromer, potentially leading to a cure.

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.

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.

First Day of a New Life' for a Boy With Sickle Cell

Kendric Cromer starts a hopeful journey free from sickle cell disease thanks to new gene therapy.

Gene Therapies Could Transform Treatment of Rare Blood Disorders - News Center

Gene therapy shows promising results in treating rare genetic blood disorders like beta thalassemia and sickle cell disease.

New Jersey college student suffering from sickle cell disease has newfound hope with gene therapy at CHOP

Gene therapy at Children's Hospital of Philadelphia offers new hope for sickle cell disease, potentially being a life-changing treatment.

First Patient Begins Newly Approved Sickle Cell Gene Therapy

Commercially approved gene therapy offers hope to sickle cell patients like Kendric Cromer, potentially leading to a cure.
moregene-therapy

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.
#healthcare

FDA approves first CRISPR therapy-here's how it works against sickle cell

The FDA has approved two gene therapies for the treatment of sickle cell disease, one of which is the first CRISPR/Cas9-based treatment to be approved in the US.
Sickle cell disease affects around 100,000 people in the US and can lead to anemia, organ damage, and early death.

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.

FDA approves first CRISPR therapy-here's how it works against sickle cell

The FDA has approved two gene therapies for the treatment of sickle cell disease, one of which is the first CRISPR/Cas9-based treatment to be approved in the US.
Sickle cell disease affects around 100,000 people in the US and can lead to anemia, organ damage, and early death.

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.
morehealthcare

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.

Innovative Thinking Could Make New Sickle Cell Treatments More Accessible

Gene therapies for sickle cell disease offer hope for functional cure, but high costs and accessibility challenges remain.

Life-changing' sickle cell drug approved for NHS roll-out after patient-led campaign

Successful patient-led appeal leads to new sickle cell treatment recommendation by NICE in the UK.

The Fight to Pay for Gene Therapy

Sickle-cell disease causes inflammation, pain, and blocked blood flow due to misshapen red blood cells.
Racism and lack of empathy in healthcare pose barriers to adequate pain management for sickle-cell patients.

New Queen's Hospital machine for disease that 'affects every aspect of life'

Spectra Optia Apheresis system benefits sickle cell patients by reducing transfusions and managing pain.
Raising awareness about sickle cell, antenatal screening, and treatments like red cell exchange are crucial for effective management.

Former Patriot Devin McCourty honored as 'Hero Among Us' at Celtics game

Devin McCourty, former Patriots safety, was honored with the Celtics' 'Hero Among Us' award for his work in the community.
McCourty and his twin brother co-founded 'Tackle Sickle Cell' over 10 years ago to raise awareness for the disease and have raised over $1.2 million for Boston Medical Center.

'I hope new test will change lives of rare blood disease sufferers like me'

Genetic test for sickle cell disease rolled out across NHS
Test helps match blood transfusions to patients with sickle cell disease

Landmark sickle cell treatment is axed by regulators

A drug approved to treat sickle cell disease has been scrapped by the UK government, despite being hailed as the first new drug in over 20 years for the condition.
The decision has sparked criticism from campaigners who argue that it leaves a reliance on only one licensed NHS treatment for sickle cell patients.

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

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.

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.

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
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.

I told my high school friend we'd battle sickle cell together. But she didn't make it past 18

Sickle cell disease is a common inherited disorder in Kenya, affecting millions globally.
[ Load more ]