Cranbury Pharmaceuticals has introduced a groundbreaking product, Emflaza® oral suspension (deflazacort), designed specifically for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder characterised by progressive muscle degeneration and weakness.
This medication is indicated for patients aged five and older, addressing a critical need in a community where treatment options are limited.
DMD predominantly affects boys, typically manifesting symptoms in early childhood. It is a rare condition, occurring in approximately 1 in 5,000 live male births globally, with an estimated 20,000 children diagnosed annually worldwide.
The FDA approval of Deflazacort oral suspension marks a significant milestone for patients, caregivers, and physicians involved in managing DMD. Deflazacort should not be used in patients who have a known allergy to deflazacort itself or any of the inactive components present in the oral suspension.
This approval underscores the importance of enhancing accessibility to effective therapies, offering hope and support for the individuals affected by this devastating disease.