Sep 10, 2019

Sickle cell persists, with no new treatments

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Data: Healthcare Cost and Utilization Project; Chart: Andrew Witherspoon/Axios

Almost 9 out of 10 hospitalizations for sickle cell disease are for black patients, and most of those patients are younger than 35, new federal data show. Three-quarters of sickle cell patients, who often experience severe pain and racial discrimination, also have government insurance.

The big picture: New treatments for sickle cell have been sparse, despite investments in other rare diseases. But Global Blood Therapeutics hopes to roll out a pill next year, pending federal approval, and Bluebird Bio is aiming to get approval for a sickle cell gene therapy by 2022.

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Gene therapies' accessibility problem

Adapted from PwC Health Research Institute; Graphic: Axios Visuals

Gene therapies are physically out of reach for many patients, on top of their cost, according to a new PwC Health Research Institute report.

Why it matters: This further exacerbates the rural-urban divide in access to care.

Go deeperArrowOct 2, 2019

Doctors struggle with the ethics of genetic testing for patients

DNA sequencing is producing vast amounts of data, but researchers and doctors often don't know what it means or what can be done about the information it reveals, the Wall Street Journal reports.

Why it matters: Providers and scientists are grappling with how much information to give patients who have had their genes scanned.

Go deeperArrowOct 7, 2019

Patients issue new pleas for experimental gene editing

Photo: Sandra Baker/Getty Images

Patients and parents of children with life-threatening diseases may start asking for experimental gene editing as a form of treatment, STAT reports.

The big picture: Bioethicists, genetic engineers and lawmakers are now asking the same ethical questions that led to the 2018 law allowing "the right to try" experimental drugs prior to FDA approval: namely, do patients have a right to try gene editing technology?

Go deeperArrowSep 26, 2019