Here’s the latest news from PTC Therapeutics about Deflazacort (EMFLAZA®). A publication of data in Muscle & Nerve comparing the efficacy and safety of deflazacort and prednisone/prednisolone from the placebo arm of the ACT DMD study has been published in Muscle & Nerve. Enjoy reading and thank you for sharing these articles within the DMD community.
The results demonstrated a clinically differentiated benefit of deflazacort over prednisone/prednisolone in slowing disease progression as measured using physical function endpoints and the time to delay loss of ambulation. Duchenne muscular dystrophy patients treated with deflazacort had notably less decline from baseline in 6-minute walk distance at Week 48 than those treated with prednisone/prednisolone.
Stuart W. Peltz, Ph.D. Chief Executive Officer of PTC Therapeutics, Inc. said:
“This publication supports the benefit of deflazacort in slowing the progression of Duchenne compared to other corticosteroids,”
“The data indicates that deflazacort should be the standard of care for all patients with Duchenne. The availability of deflazacort, a treatment that has the potential to alter the natural history of Duchenne, supports the need for early diagnosis in patients with this disease.”
What is Deflazacort
Deflazacort is a corticosteroid that works through receptors in the body to effectively inhibit inflammation.
Clinical studies demonstrated that Emflaza:
- delayed the loss of muscle strength of Duchenne patients
- improved the ability to accomplish tasks related to standing, climbing stairs, to run or walk 30 feet
- Slowed the loss of muscle strength over time
More on Emflaza.com
Access Deflazacort in Canada
In Canada, eligible patients can ask to receive deflazacort through a Special Access Program (SAP). This Health Canada program enables doctors to prescribe a drug not approved for sale or distribution in Canada to patients with a severe or life-threatening disease that has no other viable treatment. Special Access Programme
About PTC Therapeutics
PTC Therapeutics was one of the first pharmaceutical companies to develop a treatment for Duchenne muscular dystrophy (DMD). Developing ataluren (Translarna™), a treatment that works on a nonsense mutation in the dystrophin gene took 20 years. When PTC started its research, there was no North Star Ambulatory Assessment (NSAA), a mobility test that measures the results of treatment. The company began with a blank sheet. Its hard work and persistence encouraged other pharmaceutical companies to jump in and investigate new therapies for DMD. More at About PTC (vloglaforcedmd.com)
More interesting link
Ptc Therapeutics announces publication data demonstrating he Clinical Differentiated Benefit of Deflazacort: News Releases (ir.ptcbio.com)
Effects of Deflazacort Versus Prednisone on Bone Mass, Body Composition, and Lipid Profile: A Randomized, Double-Blind Study in Kidney Transplant Patients: Effects of Deflazacort Versus Prednisone (academic.oup.com)
A Comparison of the Effects of Deflazacort and Prednisone Versus Placebo on Timed Functional Tests in Boys with Duchenne Muscular Dystrophy: A Comparison of the Effects of Deflazacort (n.neurology.org)
