There are approximately 7,000 different types of rare disease and researchers estimate that there are more than 300 million people worldwide living with such a condition, according to the National ...
“There are different types of registries used based on needs ... Recently, the National Organization for Rare Disorders (NORD) issued a joint declaration of 10 key principles for rare ...
"Project Baby Lion" is the first step in growing the practice of genomic medicine on Long Island and within New York State.
Key findings from 2024 focused on conditions like vaccine-induced immune thrombotic thrombocytopenia, immune thrombocytopenia ...
MADISON, Wis. (WBAY) - Rare diseases impact one out of every 10 Americans, but a voucher to incentivize drug companies to research rare disease treatments expired earlier this month after being ...
Gene therapies are revolutionizing treatment for hemophilia A and sickle cell disease; however, they are saddled with hefty ...
Ionis won FDA approval for a drug for patients with a deadly genetic disease that impedes the body’s ability to break down ...
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