New PhRMA Report: Medicines in development for patients with rare diseases demonstrate the promise of innovation

Today, there are 7,000 known rare diseases, or diseases that affect fewer than 200,000 people in the United States. But those suffering from the impact of these diseases have more hope today because of advances in science that promise brighter futures for American patients and families. A new report, “Medicines in Development for Rare Diseases,” offers insight into the more than 560 novel treatments currently in the biopharmaceutical pipeline for the 30 million Americans currently living with a rare disease that have few or no treatment options, including patients with multiple myeloma, cystic fibrosis, amyotrophic lateral sclerosis (ALS) and enzyme deficiency disorders.
Source: The Catalyst - Category: Pharmaceuticals Authors: Tags: Medicines in Development ALS Rare Diseases Source Type: news