Robin Alderman faces a painful truth: Gene therapy can cure her son Camden’s rare, inherited immune deficiency. But this is not available to him. London-based Orchard Therapeutics stopped investing in an experimental treatment for Wiskott-Aldrich syndrome in 2022. And there are no gene therapy studies for him to participate in. “We feel like we’ve been… Read More »
The promise of gene therapy looms large for families dealing with rare genetic disorders. These types of treatments offer one-time treatment. But families and researchers worry that such treatments may be out of reach. Collectively, approximately 350 million people worldwide suffer from rare diseases, most of which are genetic. But each of the 7,000 individual… Read More »
Scientists have genetically engineered a new species of zebrafish with transparent skin to allow full observation of their internal organs – Richard White/Boston Children’s Hospital At the heart of Doha’s Education City, a vast, sun-bleached campus of tech companies and universities, lies a healthcare facility that Qatar hopes will lead the way in personalized medicine… Read More »
Prescription drugs and vaccines have revolutionized healthcare, significantly reducing deaths from disease and improving quality of life worldwide. So how do researchers, universities, hospitals, and the pharmaceutical industry decide which diseases to try to develop drugs for? Working as director of the Health Outcomes, Policy, and Evidence Synthesis group at the University of Connecticut School… Read More »
Emerging social problems or opportunities can significantly impact the funding available for the development of drugs for specific diseases. When COVID-19 ravaged the world, funding from Operation Warp Speed led to vaccine development in record time. Public awareness campaigns, such as the ALS ice bucket challenge, can also directly raise money for research. This viral… Read More »