Deflazacort over prednisone/prednisolone?

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)

A call to resilience

A text written by Guylaine Rivard
This is a text that has been originally published in the group Les mamans Duchenne.

On June 18th in 2009, it’s been almost ten years ago today that the DMD diagnosis for Félix fell on us. I say dropped on us, but it was more or so as if I got shot in my chest. Duchenne muscular dystrophy…pow! Incurable…pow! Loss of walking ability, decreasing life expectancy, wheelchair…Pow! Pow! Pow!


I am rolling with the punches. Each word, I feel like I’m taking a step back with my chair. I can’t; I really can’t believe that such a thing is happening to us…

This incurable disease will slowly take my little boy away without letting me do anything to stop it. I went shopping with Félix right away after the diagnosis because that’s what we planned to do: going for dinner at a restaurant and buying him sneakers.

I can remember sitting at a table, Félix couldn’t stop talking (as usual). I look at him, and I smile. He’s six years old. I just want to scream and cry.


First clinic appointment

There comes his first neuromuscular clinic. I’m waiting for our turn in the corridor. I feel devastated. I see a family with a youngster who was probably 17-18 years old or so. The parents are talking to everybody:

” Hi, Daniel! How are you?”  The mother speaks with interveners. Between two words, she gives pieces of a cookie to her son, because he can’t raise his arms to his mouth. She keeps going as she still speaks, she puts the juice with a drinking straw close to his mouth. Let me say I absolutely do not understand how they’re doing to be happy talking here, and even laughing! I find them impressive, and his mom fascinates me, I don’t know how she does it.


Learning how to live with the disease

Today, I know. I know that you do learn to live with the disease. It becomes a part of our life. One day, I wake up, and resilience shows up. I got to prepare myself to deal with the disease because the diagnosis is a shock that we must go through, but other shocks will follow. I must decide to fight, to roll up my sleeves and move ahead to make sure Félix lack nothing and help him the best I can so he can also go through shocks and help him grieve.


Resilience and resistance

Resilience, resistance, I am not quite sure… But one thing is sure, we are fighting, always. (Félix’s dad is a great history fan. He made me realize that June 18th is a date that we will never forget.)

June 18th, 1940: De Gaulle’s appeal for resistance General de Gaulle calls every Frenchmen and Frenchwomen to stand up and fight against the enemy:

“But has the last word been said? Must hope to disappear? Is defeat final? No! Whatever happens, the flame of the French resistance must not be extinguished and will not be extinguished.”

I invite you, mothers, fathers and Duchenne youngsters to not give up:

“Whatever happens, the flame of the Duchenne families’ resistance must not be extinguished and will not be extinguished.”


Interesting links

This text has been published on a private group called “Les Mamans Duchenne” that is administered by Carole Auclair.