US EditionGenetic medicine can leave people with rare mutations behind. But there's new hope
- Patients with rare genetic mutations of cystic fibrosis have fewer treatment options than those with common mutations, as noted by researchers discussing mutation-agnostic gene therapies.
- Kramer-Golinkoff is diagnosed with cystic fibrosis due to a rare mutation, which current treatments cannot address, affecting 40,000 Americans diagnosed with the disease overall.
- Gene therapy trials at Columbia University aim to improve lung function and overall health, providing hope for patients with cystic fibrosis.
- Despite worsening health, Kramer-Golinkoff expresses optimism about future therapies, noting that having trials underway provides hope for those with cystic fibrosis.
46 Articles
46 Articles
Los Angeles Times
Independent EspañolGenetic medicine can leave behind people with rare mutations, but there are hopes
Emily Kramer-Golinkoff doesn't get enough oxygen with every breath.
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Associated Press News
Genetic medicine can leave people with rare mutations behind. But there's new hope
People with diseases caused by rare mutations have fewer options and poorer prospects than other patients despite rapid recent advancements in the growing field of genetic science.
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