Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for ...
When our son Peter was diagnosed with an ultra-rare form of muscular dystrophy at the age of 10, the first question we asked was: Is there a treatment? The answer was no. By the time our daughter ...
Median net sales for the 19 drugs in 2022 were $1.97 billion (range, $988 million-$8.36 billion). The drugs with the highest net sales were lenalidomide (Revlimid; $8.36 billion), ...
The Food and Drug Administration’s rare pediatric disease priority review voucher program, which has been providing incentives for lifesaving innovations since 2012, is doomed to disappear unless ...
Rare diseases reveal that our sense of identity rests on fragile biological infrastructure, much of it operating outside ...
US regulators laid out new guidelines for approving custom-made treatments for individual patients, a move that could bring ...
Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...
The global pharmaceutical industry is make huge investments in treatments for rare diseases, which once drew little interest from major drugmakers. A look at some key statistics on rare diseases: ...
Peer-reviewed publication examines the benefits of proactive exome reanalysis on diagnostic outcomes Ambry Genetics’ Patient for Life program is supported by a dedicated team of scientists who ...
Seles’ first symptoms appeared suddenly around three years ago. She began having double vision and weakness in her arms and ...
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