Emily Mwaka doesn’t like speculating about the next pope, especially on the color of his skin. So when the head of the Catholic laity in Kampala, Uganda, recently came upon a small group of Christians discussing a newspaper article about possible papal contenders — including some from Africa — she asked them to stop it.
For example, comprehensive data about cystic fibrosis is scarce among African populations – affecting people who live on the continent as well as those who trace their ancestry there.Black cystic fibrosis patients are more likely than their white counterparts to be among the 10% who don’t benefit from modulator therapies.
While there’s little chance of changing market dynamics, researchers said, one solution is to develop “mutation agnostic” gene therapies targeting all patients with a disease. This approach is being tried in diseases of the retina as well as cystic fibrosis.“There’s a huge push to develop these therapies,” said Dr. Garry Cutting of the Johns Hopkins Cystic Fibrosis Center.Most of the 14 experimental gene therapies in the
for the disease aim to help patients with any mutation, the Cystic Fibrosis Foundation says, delivering a new, correct version of the CFTR gene to cells. Getting correct copies of the CFTR gene would enable cells to make normal proteins no matter what mutation causes a patient to have no, or not enough, functional CFTR proteins.One treatment, partially funded by the foundation, is sponsored by Spirovant Sciences, a company Emily’s Entourage provided seed money to launch. The first patient received the therapy in November in a 53-week clinical trial at Columbia University that aims to determine if it’s safe and how long it stays in the lung.
Kramer-Golinkoff said she’s more optimistic about her future these days, even as her own illness worsens. At this point, she’s living with 30% lung function, suffers from kidney issues and has high blood pressure in her lungs. She depends on insulin for her diabetes and takes numerous pills daily.
“You have to make really conscientious choices ... throughout the day on how to use your limited energy. And that’s really difficult to do when you have big dreams and important work and life to live,” she said.This time, Senate Republicans are advancing the effort after President Donald Trump declared his intent to “keep men out of women’s sports.” and made it a major
in last year’s election, dividing Democrats on how to respond.The bill applies to participation in girls’ and women’s sports that are sponsored by public schools, public universities and publicly chartered community colleges.
It also prohibits any sort of government agency or athletic association from investigating or punishing a school or higher education institution for maintaining separate sports teams for girls or women.For well over an hour, Republicans and Democrats debated the bill, at times hotly. The sponsor, Sen. Judy Ward, a Republican from Blair County, said the bill would “ensure all young women have a fair chance to compete in the sports they love.”