Accelerating Rare Disease Drug Development: Lessons Learned from Muscular Dystrophy Patient Advocacy Groups.

With scientific and molecular advancements related to disease pathogenesis, advances in gene and stem cell therapies, and the promise of lucrative markets for biopharmaceutical companies, there has been a rapid expansion in the number of potential new muscular dystrophy (MD) treatments. The first champion for a newly diagnosed MD patient and their caregivers is typically an MD-specific patient advocacy group (PAG). Muscular dystrophy PAGs have been among the most active in the rare disease drug development space.

Clinical trials assessing ototopical agents in the treatment of pain associated with acute otitis media in children.

Otalgia (ear pain) is one of the characteristic symptoms and best predictor of acute otitis media (AOM) in children. Although oral pain medications are the current mainstay for the treatment of AOM-associated otalgia, ototopical agents have been investigated as an alternative treatment strategy. To permit review and assessment of this treatment modality, a systematic literature search was conducted to identify all randomized, controlled trials of ototopical agents.