When it comes to preserving muscle strength and function,

IT’S NOT JUST A FIBER. IT’S HIS FUTURE.

CAMERON IS A 3-YEAR-OLD, NEWLY DIAGNOSED DUCHENNE PATIENT

Cameron demonstrated developmental difficulties, and his parents noticed that he fell often and used his hands and arms to climb up his body to get from squatting to standing (known as Gowers’ sign).

After his Duchenne diagnosis, Cameron’s family was devastated and worried about the future.

DELAY HIS DISEASE PROGRESSION WITH EMFLAZA®

EMFLAZA is now approved to treat Duchenne in children 2 years of age and older1

It’s important to detect and treat Duchenne as early as possible2,3

  • Once muscle is lost, it cannot be restored
  • Timely treatment and management interventions are critical to help maintain muscle function for as long as possible

EMFLAZA can help minimize the challenges of Duchenne3,4

SIGN UP TO LEARN MORE

EMFLAZA IS PROVEN TO PRESERVE MUSCLE FUNCTION LONGER THAN PREDNISONE4,a

  • After 12 weeks of treatment, EMFLAZA 0.9 mg/kg/day was shown to significantly improve muscle strength compared with placebo (0.15 vs -0.10, P=0.017)1
Change in average muscle strength score (intention-to-treat population)4

STUDY DESIGN

A phase 3, multicenter, randomized, double-blind, placebo-controlled, 52-week study. Safety was evaluated by the incidence of adverse events, changes in clinical laboratory findings or physical examinations, and vital signs. Comparisons between deflazacort and prednisone are not included in the approved Prescribing Information for deflazacort. This study did not allow for long-term follow-up to observe the effect of steroids over longer periods of time. Patients on intermittent prednisone regimens may have had fewer adverse events than daily prednisone dosing.

The primary efficacy end point was average change in muscle strength from baseline to Week 12 compared with placebo, as assessed by modified Medical Research Council (MRC) scale, an 11-point scale. A total of 18 muscle groups were tested for each patient.

Secondary end points included change in average muscle strength from Week 12 to 52 and pulmonary function testing. Additional end points included timed functional testing: standing from lying position, climbing 4 stairs, running/walking 30 feet, and propelling a wheelchair 30 feet.
Full Study Summary

EMFLAZA offers a proven safety profile

EMFLAZA PROVIDES A FAVORABLE TOLERABILITY PROFILE1

References: 1. Emflaza [package insert]. South Plainfield, NJ: PTC Therapeutics, Inc; 2019. 2. Blake DJ, Weir A, Newey SE, Davies KE. Function and genetics of dystrophin and dystrophin-related proteins in muscle. Physiol Rev. 2002;82(2):291-329. 3. Shieh PB, Mcintosh J, Jin F, et al; ACT DMD Study Group. Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post hoc analysis from the ACT DMD trial. Muscle Nerve. 2018;58(5):639-645. 4. Griggs RC, Miller JP, Greenberg CR, et al. Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology. 2016;87(20):2123-2131.