How to Successfully Launch a Rare Disease Drug in a Patient-Centric World

The Impact of Rare Diseases on the Experience of Siblings

FDA Panel Backs Expanded Use of J&J and Bristol Myers' CAR-T Cancer Therapies

Genetic Treatment for Sickle Cell Anemia: A New Dawn

Diagnosing Rare Diseases: Challenges and Technological Solutions

Deciphering FDA Drug Approval Designations: A Practical Guide

Aprocitentan: Breakthrough Treatment for Resistant Hypertension Approved by FDA

CA2 Bio Leads Innovation in Oncology with A2B530, an Advanced Cell Therapy Offering Targeted Treatment for Colorectal Cancer

Comparative Analysis of Orphan Drug Policies on an International Level

Regulatory Framework for Orphan Drug Designation: Pharmaceuticals and Biologics