Sickle Cell Disease Study Testimonials

My Contribution to Clinical Studies

3 mins WATCH NOW

Ada Gonzalez lives with sickle cell disease and enrolled in a clinical study decades ago that she benefits from today.

Making an Informed Decision As a Family

4 mins WATCH NOW

Jewel is a teenager living with sickle cell disease. She and her mother, Paula make informed decision about participating in clinical studies.


LentiGlobin Gene Therapy Shows Promise for Severe Sickle Cell Disease

February 22, 2020

LentiGlobin gene therapy led to substantial reductions of sickle hemoglobin and considerable improvement in key markers of hemolysis.

A Young Mississippi Woman's Journey Through A Pioneering Gene-Editing Experiment

December 25, 2019

Doctors removed bone marrow cells from Gray's body, edited a gene inside them with CRISPR and infused the modified cells back into her system this summer. And it appears the cells are doing what scientists hoped — producing a protein that could alleviate the worst complications of sickle cell.

FDA Approves Novel Treatment to Target Abnormality in
Sickle Cell Disease

November 25, 2019

With Oxbryta, sickle cells are less likely to bind together and form the sickle shape, which can cause low hemoglobin levels due to red blood cell destruction. This therapy provides a new treatment option for patients with this serious and life-threatening condition.

Gene-Edited 'Supercells' Make Progress In Fight Against Sickle Cell Disease

November 19, 2019

Doctors are reporting the first evidence that genetically edited cells could offer a safe way to treat sickle cell disease, a devastating, incurable disorder that afflicts millions of people around the world.

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