What's Happening

November 2017

November 2017



Glutamine and Sickle Cell Anemia

Yutaka Niihara, MD, MPH, chief scientific officer and founder of Emmaus Life Sciences talks about their orphan designated drug L-glutamine to treat patients with sickle cell anemia.



Sickle Cell Patients Endure Discrimination, Poor Care And Shortened Lives
November 4, 2017

Click here to read the companion article...

Lakiea Bailey, Ph.D., 39, Patient, Director of the Sickle Cell Community Consortium, Research Scientist
September 4, 2017

Angela G. King

Her story: At age 5, Lakiea Bailey experienced the first sickle cell crisis that landed her in a hospital, and since then she's endured hundreds of hospitalizations, blood transfusions, and surgical procedures. Yet she went on to earn a bachelor's degree in biochemistry and molecular biology, and a doctorate in molecular hematology and regenerative medicine. Now, in addition to numerous other volunteer efforts, she is director of a consortium of nonprofit organizations aimed at empowering SCD patients and their caregivers to have a leadership voice in research, policy advocacy, and education.
Click here to read full article...

Gene therapy lets a French teen dodge sickle cell disease
Associated Press | Big Story
Marilynn Marchione
March 1, 2017

Sickled Red Blood Cells

A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.

The first-in-the-world case is detailed in Thursday's New England Journal of Medicine

Is gene therapy available to treat my disorder?
Genetic Home Reference | National Institute of Health
March 1, 2017

GHR Logo

Gene therapy is currently available only in a research setting. The U.S. Food and Drug Administration (FDA) has not yet approved any gene therapy products for sale in the United States.

Hundreds of research studies (clinical trials) are under way to test gene therapy as a treatment for genetic conditions, cancer, and HIV/AIDS. If you are interested in participating in a clinical trial, talk with your doctor or a genetics professional about how to participate.

Tainted Blood
Huffington Post
Angela G. King
October 17, 2016

Angela G. King

One of the most common genetic disruptions to blood flow in the human body that exists, Sickle Cell Disease afflicts millions of men, women and children around the globe - up to 100,000 of them living in the United States. Yet a universal cure, or even slate of treatments to effectively combat its often painful and debilitating, at times fatal symptoms remain elusive. That could finally be about to change substantively. In her three-part series, she presents stories that explores this prospect among a community still languishing so far beneath the radar of public consciousness.

Couple's attempt to do the
right thing brings more grief

Los Angeles Times
Steve Lopez
March 11, 2009
Read the article...


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