Buried within the One Big Beautiful Bill Act is a provision that could improve or even save the lives of the 30 million ...

As one FDA initiative works to sustain the momentum of rare disease research, other events threaten to slow progress.

ImCheck’s ICT01 Receives FDA Orphan Drug Designation for Treatment of Acute Myeloid Leukemia Clinical data showing ...

Targeted Oncology connects oncology professionals with updates on immunotherapy, biomarkers, cancer pathways, and targeted ...

Avadel Pharmaceuticals announces Orphan Drug Designation for LUMRYZ to treat Idiopathic Hypersomnia, emphasizing its once-nightly dosing benefit.

Retail chatter around Klotho Neurosciences picked up late Thursday after the company said that the FDA had granted Orphan Drug Designation to its ALS gene therapy candidate, KLTO-202. The designation ...

The FDA's Orphan Drug Designation is granted to investigational therapies intended for the treatment of rare diseases or conditions that affect fewer than 200,000 people in the United States.

IASO Bio possesses comprehensive capabilities spanning the entire drug development process, ... Equecabtagene Autoleucel (Fucaso), has been granted Orphan Drug Designation ...

ATSN-201 received orphan drug designation from the FDA for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics.

*Research on the supply guarantee system of orphan drugs in South Korea from the perspective of accessibility and its enlightenment. YANG Yifan, XlE Jinping, WENG Tingting, YAN Liyu, SHAO Rong ...