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Dr. Doris Wethers Blazed a Trail for Newborn Testing for Sickle Cell Disease

Sickle Cell Disease Association of America, Inc. (SCDAA) celebrates the life and work of Dr. Doris L. Wethers who died at the age of 91 on January 28, 2019.
Dr. Wethers’ research and advocacy efforts helped lead to mandatory testing of all newborns for sickle cell disease. She broke racial barriers in the medical world when she became the first black chief of a medical department in 1965 as the director of pediatrics at Knickerbocker Hospital in West Harlem. She was later director of pediatrics from 1969 to 1974 at Sydenham Hospital (which was shuttered in 1980) and then, until 1979, at St. Luke’s Hospital Center (now Mount Sinai St. Luke’s). She became St. Luke’s first black attending physician in 1958.
Dr. Wethers opened sickle cell disease programs at all three hospitals, conducted research and helped draft landmark legislation in New York to require screening of infants for the disorder. Over the course of her career at the hospitals, the average life expectancy of children born with sickle cell rose from about 18 to 50. The increase was attributed largely to early detection, infection prevention through the use of penicillin and other breakthroughs that helped mitigate pain and prolong life.
In 1987, Dr. Wethers was the chair of a National Institutes of Health panel that recommended routine testing for newborn babies regardless of race or ethnicity. New York was the first state to mandate such testing, in 1975, and all states provided for universal screening by 2006.
SCDAA thanks Dr. Wethers for being a trailblazer for women and for people of color in the medical field and for her tireless efforts, as a true pioneer, to serve those living with sickle cell disease. Rest in peace, Dr. Wethers, knowing that your work advanced sickle cell treatment and helped to improve the quality of life for those living with this life-threatening disease. SCDAA sends its condolences to Dr. Wethers’ family and friends. You are in our thoughts and prayers.
(This statement includes excerpts from the February 7, 2019 New York Times article,
“Dr. Doris Wethers, 91, on Front Lines Against Sickle Cell, Dies.”) Photo credit: Courtesy of the New York Times.

 

Emmaus Life Sciences Launches Its Commercial Co-Payment Assistance Program for Endari™

TORRANCE, Calif., Jan. 28, 2019 /PRNewswire/ — Emmaus Life Sciences, Inc. (Emmaus), a leader in sickle cell disease treatment, announced today that it will provide financial assistance to help eligible patients afford their monthly co-payment for Endari™ (L-glutamine oral powder) [1] . The program is limited to financially eligible patients covered by commercial insurance.

A significant number of individuals with sickle cell disease are covered by commercial insurance. In some cases, patients may have difficulty affording the monthly co-payment amount.
Mark Diamond, Emmaus’ Vice President of Commercialization, commented: “We are committed to removing barriers between patients and Endari – giving a greater number of patients access to our effective treatment for sickle cell disease.”
Click here to read the full press release.
Sickle Cell Disease Association of America, Inc. (SCDAA) in no way endorses any medications, treatments, clinical trials, or studies reported through Get Connected. Information is provided to keep the readers informed. Because the manifestations and severity of sickle cell vary among individuals, personalized medical management is essential. Therefore, it is strongly recommended that all drugs and treatments be discussed with the reader’s physician(s) for proper evaluation and treatment.

 

CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease

CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the treatment of sickle cell disease (SCD). CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies.

The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.  A drug granted Fast Track Designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if relevant criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a Biologics License Application (BLA).

In October 2018, CRISPR and Vertex announced the FDA acceptance of the Investigational New Drug application (IND) for CTX001 for the treatment of SCD, and enrollment in a Phase 1/2 trial in SCD is currently underway in the U.S. The companies are also evaluating CTX001 for the treatment of β-thalassemia, and enrollment in a Phase 1/2 trial in β-thalassemia is currently open at multiple clinical trial sites in Europe.

Click here to read the press release issued today.