Gene therapy may cure rare diseases. But drug dealers have few incentives, leaving families desperate
by Robin Alderman is faced with a painful reality: gene therapy could cure her son Camden’s immune deficiency. But it is not available to him. In …
Tag: rare diseases
Takeaways from AP report on access to gene therapies for rare diseases
The promise of gene therapy is huge for families dealing with rare genetic disorders. Such treatments offer the possibility of one-time cures. But families and …
Why genetically modified fish are being bred to fight rare childhood diseases
Scientists have genetically engineered a breed of zebrafish with transparent skin to precisely observe its internal organs – Richard White/Children’s Hospital Boston At the heart …
CRISPR and other new technologies open doors to drug development, but which diseases are prioritized? It comes down to money and science
Emerging social issues or opportunities can significantly affect the funding available to develop drugs for certain diseases. When COVID-19 swept the world, funding from Operation …
CRISPR and other new technologies open doors to drug development, but which diseases are prioritized? It comes down to money and science
Prescription drugs and vaccines have revolutionized healthcare, dramatically reducing death from disease and improving quality of life around the globe. But how do researchers, universities …