Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are ...

Kiniksa stock rocketed 20% after first-quarter results on April 29. The biotech stock then broke out of a cup base with a buy ...

Expansion of the Medicare drug price negotiation rare disease drug carve-out would unnecessarily limit the number of drugs ...

The Inflation Reduction Act includes an exemption for orphan drugs for a single indication, but experts say this is far from ...

Paris: Sanofi has received orphan drug designation from the Ministry of Health, Labour and Welfare (MHLW) in Japan for ...

Aardvark's lead drug ARD-101 targets PWS and HO with trials underway and funding secured through 2027 after a $94.2 million ...

The Orphan Cures Act has been added back into the Senate version of President Donald Trump’s “big, beautiful bill” after being ...

7 years of market exclusivity in the U.S. upon FDA approval ...

Japanese MHLW grants orphan drug designation to Sanofi’s riliprubart for chronic inflammatory demyelinating polyneuropathy: Paris Tuesday, July 1, 2025, 09:00 Hrs [IST] The Mini ...

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 ...

Riliprubart granted orphan drug designation in Japan for chronic inflammatory demyelinating polyneuropathy. Two phase 3 studies are currently underway testing riliprubart in peopl ...

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 ...