The drug, discovered by Japan Tobacco and once in the hands of Merck, was licensed by BridgeBio in 2019.
A once-rare disease that causes a person to develop a severe allergy to red meat is seeing an “explosive rise” across America ...
PAP is a rare lung condition that can be difficult to diagnose because it may mimic many other more common lung diseases,” ...
Us Orphan Drug Rare Disease Market Offers US$ 190 Billion Opportunity Highlights Kuick ResearchDelhi, Oct. 31, 2025 (GLOBE ...
Researchers at Queen Elizabeth Hospital Birmingham (QEHB), a leading centre for the PROCLIPI Study, have contributed to new ...
When Bree Whitelegg’s son fell dangerously ill, her simple question to a doctor led to a two-year investigation into infant ...
Rare disease drug development presents unique challenges: patient populations are often minimal, clinical endpoints may not be well-established and traditional approaches to evidence generation cannot ...
A young boy battling a rare disease saw the Kansas City community come together ... Tucker, 3, is unable to participate in ...
Health Secretary Robert F. Kennedy Jr. called the treatment 'a powerful glimpse into the future of personalized medicine' ...
Researchers at the VIB-UAntwerp Center for Molecular Neurology have visualized how brain network development is altered in a ...
Leiden researchers have found clues to why a rare pregnancy disorder is mild in some babies but life-threatening in others.
From hard-to-find mushrooms to unique coffee beans to certain delicacies, this list includes some of the world's rarest ...