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 ...

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 ...

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 ...

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

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

ABBV-706 is an ADC targeting SEZ6 developed by AbbVie. It is linked to a topoisomerase 1 inhibitor (TOP1i) via a ...

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 ...

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