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A provision protecting expensive medications, used to treat multiple rare diseases, from the Medicare drug price negotiations ...
The Inflation Reduction Act includes an exemption for orphan drugs for a single indication, but experts say this is far from ...
The Orphan Cures Act has been added back into the Senate version of President Donald Trump’s “big, beautiful bill” after being ...
The pharmaceutical industry’s lobbying muscle scored a high-stakes victory on Monday when the Senate parliamentarian reversed a previous decision and ruled that the Optimizing Research Progress Hope ...
Hemispherian’s GLIX1 to treat malignant glioma receives US FDA orphan drug designation: Oslo, Norway Thursday, July 3, 2025, 15:00 Hrs [IST] Hemispherian AS, an Oslo-based pharm ...
Paris: Sanofi has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to Riliprubart ...
Riliprubart is an immunoglobulin G4 humanized antibody that is designed to selectively inhibit activated C1s in the classical complement pathway of the innate immune system.
ABBV-706 is an ADC targeting SEZ6 developed by AbbVie. It is linked to a topoisomerase 1 inhibitor (TOP1i) via a ...
Congenital adrenal hyperplasia (CAH) is a rare autosomal recessive disease1 with an estimated global incidence of approximately 1 in 14,000–18,000 live births2A phase II trial has recently been ...
Paris: Sanofi has received orphan drug designation from the Ministry of Health, Labour and Welfare (MHLW) in Japan for ...
Sanofi (ENXTPA:SAN) recently received orphan drug designation from Japan's Ministry of Health for riliprubart, aimed at treating chronic inflammatory demyelinating polyneuropathy. This underscores the ...
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