Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A
Winrevair is the first activin signaling inhibitor therapy for PAH approved in Europe
It is the first targeted fluorescent imaging agent that illuminates ovarian cancer intraoperatively, enabling the detection of more cancer for removal
The trial met the primary endpoint of overall survival with a single priming dose of tremelimumab plus Imfinzi every four weeks vs. sorafenib
Extension of hypericin orphan designation beyond cutaneous T-cell lymphoma
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