For Americans living with rare diseases, the Rare Pediatric Disease Priority Review Voucher Program is a vital tool. Yet its reauthorization is at risk.
Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...
The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...
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 ...
The UK has the science base and ambition to transform outcomes for people living with rare diseases. Delivery now depends on aligning science, ...
Rare Disease Day raises awareness for the thousands of rare diseases affecting millions. Advancements in technology, such as gene therapy, offer hope for potential cures for rare diseases in the ...
A rare disease or disorder is defined as affecting fewer than 200,000 Americans each year. There are more than 7,000 different rare disorders. Combined, 1 in 10 Americans has a rare disorder, so the ...
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