Brightpath Biotherapeutics Co. Ltd.’s iPS cell-derived BCMA CAR-natural killer T cell therapy candidate has been awarded orphan drug designation by the FDA for the treatment of multiple myeloma.

Clinical-stage oncology company Prescient Therapeutics (ASX: PTX) is looking to disrupt a US$1.8 billion market on the back ...

The GOP tax bill repeals the Inflation Reduction Act’s price controls for drugs for rare diseases.

Sanofi (NASDAQ: SNY) is one of the 10 Best Dip Stocks to Buy According to Billionaires. On June 25, Sanofi (NASDAQ:SNY) ...

Senators made the right decision for America's most vulnerable patients ― now, the House must follow suit. The ORPHAN Cures Act is a simple fix to a costly mistake ― and a necessary step to keep rare ...

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 ...

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

Aldeyra's Reproxalap progresses towards FDA approval, with a potential $100M AbbVie deal. There’s still upside potential, ...

An AI-enabled drug discovery approach identified a potentially game-changing treatment for Rett syndrome, which has been ...

The FDA approved Lynozyfic for relapsed multiple myeloma, but serious risks require restricted use under a special safety program.

AI-enabled drug discovery approach identified potentially game-changing treatment which has been advanced from the lab bench to an FDA Orphan Drug ...