A new AI model called popEVE can predict how likely each variant in a patient’s genome is to cause disease. The team is ...
The Orphan Drug Act has catalyzed innovation in rare disease treatments, but the law’s shortcomings — particularly around ...
Minimum volume thresholds for rare diseases: insights through the transfer of evidence IQWiG presents a method for assessing the relationship between the volume of healthcare interventions and the ...
Acute hepatic porphyria (AHP) is a rare genetic disease with symptoms that overlap with many other conditions, making it extremely challenging to diagnose. Its symptoms mostly affect women with severe ...
But in genetics, zebras are everywhere and have become the symbol for rare diseases. According to the National Institutes of Health, more than 7,000 rare diseases have been identified, with almost 80% ...
After being ousted from the top spot last year, Roche has clawed its way back to dominance among rare disease patient groups’ assessments of drugmaker reputations. PatientView compiled the views of a ...
The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive advocate for the development of rare-disease drugs. When I joined the FDA to be ...
A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a wholesale price of $4.25 million, its manufacturer announced Wednesday, making it the world’s most ...
Brightline West celebrates groundbreaking, future rail travel in Las Vegas. Brightline West breaks ground on its future Las Vegas station and rail system to California. We look at the impact high ...
When Casey McPherson became a father, his purpose became clear. He needed to raise his daughter, Rose, to be loving, courageous, healthy and strong. He needed to protect her. Many parents can ...
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