PTC Therapeutics receives FDA approval for the expansion of the Emflaza (deflazacort) labeling to include patients 2-5 years of age

PTC Therapeutics

7 June 2019 - Emflaza is the only approved treatment for all patients two and over living with Duchenne muscular dystrophy.

PTC Therapeutics today announced that the U.S. FDA approved the company's supplemental new drug application for Emflaza (deflazacort) to expand its labelling to include patients with Duchenne muscular dystrophy who are between 2- and 5-years-old. Duchenne is a rare childhood genetic disorder that causes progressive irreversible muscle deterioration and weakness. 

Emflaza was first approved by the FDA in February 2017 for the treatment of Duchenne in patients 5-years and older.

Read PTC Therapeutics press release


Michael Wonder

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Michael Wonder

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Outcome , Medicine , US , Paediatrics