Rare Disease Day is held on the last day of February to raise awareness for rare diseases and address drug development ...

The approval was based on a cohort of treatment-naïve patients in the Beamion LUNG-1 trial who had unresectable or metastatic, non-squamous NSCLC with HER2 tyrosine kinase domain mutations.

Sanofi and Regeneron’s Dupixent recommended for EU approval to treat chronic spontaneous urticaria in young children with ongoing symptoms despite treatment ...

Boehringer Ingelheim has become the second company to get a product approved under the FDA's national priority voucher ...

'They were trying to help a child who was struggling in ways none of us fully understood at the time. I never shared this to create a narrative of family conflict,' she wrote.

Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...

Lawmakers will require people to show IDs to use Indiana’s needle exchange programs under legislation heading to Gov. Mike Braun’s desk.

Neighborhood groups are mobilizing against any proposal that would bring supportive housing and addiction treatment to the campus.

Pharmaceutical costs. U.S. employers are expecting an 11 to 12% increase in pharmacy costs in 2026, which outpaces the percentage gain in general healthcare costs projected. GLP-1s contribute to this, ...

The first and only approved achondroplasia therapy to provide continuous systemic exposure to CNP over the weekly dosing intervalCommercial availability expected during early part of Q2 2026Rare Pedia ...