Amphix Bio, a pre-clinical stage company developing a new therapeutic modality for regenerative medicine, received an Orphan ...

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

STOCKHOLM, SE / ACCESS Newswire / July 15, 2025 / AlzeCure Pharma (STO:ALZCUR) (FRA:AC6) AlzeCure Pharma AB (publ) (FN ...

The FDA designates SH-110 as an orphan drug, offering a safer oral treatment option for glioma patients with swallowing ...

“Dancing molecules,” the promising new treatment for acute spinal cord injuries developed at Northwestern University, has received Orphan Drug Designation from the US Food and Drug Administration (FDA ...

KLTO‑202 is an experimental gene therapy that would deliver the instructions for a protein that may reduce inflammation and ...

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral ...

President Donald Trump’s One Big Beautiful Bill, signed into law last week, reintroduces broader exemptions for orphan drugs ...

Arcus Biosciences' cancer drug candidate, quemliclustat, earns FDA orphan drug status, ensuring incentives and exclusivity.

FDA designates FF-10832 as an orphan drug for biliary tract cancer, promising enhanced treatment options for this aggressive ...

ADRX-0405 is uniquely being tested during its 1a/b clinical trials. ADRX-0405 is labeled as a STEAP1 ADC (primarily ...