Deep breaths in and out. Something many of us take for granted. But for people with cystic fibrosis, the lungs fill with thick mucus, making it tough to get air mucus. Also clogs the pancreas and intestines, cystic fibrosis expert, Dr Marie Egan is studying ways to help the body repair the mutated DNA that causes the condition we could cure you. Doctor Egan and her Yale colleagues developed *** new gene editing technique that targets *** mutation in the CTR gene. They've designed small molecules that can be delivered into the body intravenously. We include *** small piece of DNA or blueprint that has the right instructions your own body can fix itself. Dr Egan says the molecules would be administered as *** one time therapy and could be *** delivery method and treatment for other conditions. I think it's imperative that we move forward and not just for CF but for all genetic disorders. I'm Marty Salt reporting.
Thanks to medical advances, cystic fibrosis is no longer an automatic qualifier for the Make-A-Wish Foundation
Updated: 5:58 AM EDT Mar 20, 2023
The Make-A-Wish Foundation has announced cystic fibrosis is no longer automatically a qualifying condition, because of "life-changing advances" in the outcome of the disease.The philanthropic foundation announced the policy change in a news release Friday. They explained beginning in January 2024, children with cystic fibrosis will only be eligible if they have "additional complications or factors that make the current situation critical."Cystic fibrosis is a lifelong disease progressive genetic disease, which causes an overabundance of mucus, trapping infections and blocking airways in the lungs. Treatment options for the disease have long been limited, and the diagnosis has been associated with shortened life spans.Video above: Researchers are studying a "gene editing" technique to help treat cystic fibrosisBut advances in the treatment of the disease have led cystic fibrosis patients to live decades longer. The current life expectancy for cystic fibrosis patients born between 2017 and 2021 is 53 years, an increase from 38 years a decade ago, according to the release. And in 2019, the FDA approved a "breakthrough" therapy that could be used by up to 90% of patients with the disease."This decision was not made lightly, and we understand it may result in some frustration and disappointment," wrote the organization. "As with all wish referrals, we will carefully consider any CF request that a family member, legal guardian, medical professional, or potential wish child believes meets our guidelines."The foundation said they will consult a team of 19 physicians from diverse backgrounds, as well as some of the more than 200 medical advisers who support their chapters, to ensure the new rules are applied equitably.The nonprofit explained they also grant wishes on a case-by-case basis for several other diseases, including some cancers, epilepsy, sickle cell disease, and heart disease.Per its website, the Make-A-Wish Foundation grants "life-changing wishes" for children between the ages of 2.5 and 18 years who are diagnosed with a critical illness "that is placing the child's life in jeopardy."
The Make-A-Wish Foundation has announced cystic fibrosis is no longer automatically a qualifying condition, because of "life-changing advances" in the outcome of the disease.
The philanthropic foundation announced the policy change in a news release Friday. They explained beginning in January 2024, children with cystic fibrosis will only be eligible if they have "additional complications or factors that make the current situation critical."
Cystic fibrosis is a lifelong disease progressive genetic disease, which causes an overabundance of mucus, trapping infections and blocking airways in the lungs. Treatment options for the disease have long been limited, and the diagnosis has been associated with shortened life spans.
Video above: Researchers are studying a "gene editing" technique to help treat cystic fibrosis
But advances in the treatment of the disease have led cystic fibrosis patients to live decades longer. The current life expectancy for cystic fibrosis patients born between 2017 and 2021 is 53 years, an increase from 38 years a decade ago, according to the release. And in 2019, the FDA approved a "breakthrough" therapy that could be used by up to 90% of patients with the disease.
"This decision was not made lightly, and we understand it may result in some frustration and disappointment," wrote the organization. "As with all wish referrals, we will carefully consider any CF request that a family member, legal guardian, medical professional, or potential wish child believes meets our guidelines."
The foundation said they will consult a team of 19 physicians from diverse backgrounds, as well as some of the more than 200 medical advisers who support their chapters, to ensure the new rules are applied equitably.
The nonprofit explained they also grant wishes on a case-by-case basis for several other diseases, including some cancers, epilepsy, sickle cell disease, and heart disease.
Per its website, the Make-A-Wish Foundation grants "life-changing wishes" for children between the ages of 2.5 and 18 years who are diagnosed with a critical illness "that is placing the child's life in jeopardy."
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