Survey for Canadian families living with Duchenne Muscular Dystrophy

Dear Duchenne Family, 

On behalf of Stand for Duchenne Canada, Jesse’s Journey and La Force DMD, thank you for your interest in participating in this survey for Canadian families living with Duchenne Muscular Dystrophy (DMD).


The purpose of this survey is to gather vital information to submit to Health Canada, describing the impact Duchenne has on the lives of those living with and caring for this rare disease. Currently, there is no way for caregivers and people with DMD to let Health Canada know this. We feel it is critical that this information is considered when therapies are under review with Health Canada. Currently, there are therapies for DMD approved in Europe and the US that have not yet been approved in Canada.

Learn more on the approval process in Canada

How are drugs approved for use in Canada?  •  The approval process for new drugs


It’s crucial for our DMD community to help Health Canada understand why we don’t have time to wait for these new treatment options to become available.



We request that you complete the survey at your earliest convenience as it will be closing on August 2, 2019. This survey should take about 30 minutes to complete. If you are a caregiver for more than one person with DMD, please complete this survey for each.

By agreeing to complete this survey, you consent to allow Stand for Duchenne, Jesse’s Journey and La Force DMD to publish a summary of all the data collected in this survey of which your personal and medical information will be anonymized. The individual information that we collect from this survey will be kept private.






In addition to this online survey, we are also conducting telephone interviews. The interview is vital for us to collect important additional information about your preferences that we would not be able to collect in this survey. All your personal information will continue to remain anonymous. If you are interested in participating in a 15-minute telephone interview and/or interested in getting more involved in advocating to ensure the DMD voice is being heard, please send an email to

On behalf of Stand for Duchenne Canada, Jesse’s Journey and La Force DMD, thank you in advance for your engagement. If you have any questions about this survey, please contact Nicola Worsfold at All conversations will remain confidential. 


Thank you for your interest in participating in this survey for Canadian families living with Duchenne Muscular Dystrophy



Stand for Duchenne  •  Jesse’s Journey •  La Force DMD




Exondys 51 slows down respiratory decline

Thanks to Bio Space, IOS Press, Science Daily for the content of this blog.

Duchenne muscular dystrophy is characterized by progressive muscle degeneration. In DMD patients, the pulmonary function becomes progressively impaired as the dystrophic process affects respiratory muscles, including the diaphragm. Strategies to arrest this severe gradual deterioration are needed to extend lives and improve quality of life.

Results of three clinical trials using eteplirsen show promising results

Sarepta, along with Harvard Medical School, The Children’s Hospital of Philadelphia, Nationwide Children’s Hospital, the Paul D. Wellstone Muscular Dystrophy Cooperative Research Center and Ohio State University published results from three trials in the Journal of Neuromuscular Diseases.

This study conducted in three clinical trials supports that Sarepta Therapeutics’ Exondys 51 (eteplirsen) slows respiratory decline in Duchenne muscular dystrophy (DMD).

The respiratory decline in patients treated with eteplirsen was significantly lower, and this was true across all stages of the disease evaluated.

As the disease progresses, patients require increasing levels of clinical treatment. Patients are at increased risk of death once this respiratory decline reaches a critical threshold.

Eteplirsen may slow the rate of respiratory decline and therefore may delay time to milestones of decrease. This may have notable positive implications on quality of life. Longer-term follow-up is needed.

Pulmonary function

The pulmonary function can be measured by assessing different parameters of lung function. As an example, the total amount of air that can be moved through the lungs after a maximal inspiration and then exhalation (forced vital capacity [FVC]). The FVC measures output of inspiratory and expiratory muscles. This is an excellent measure of respiratory function reserve and is widely used in DMD to assess respiratory function.

About Exondys 51

  • Exondys 51 was approved in the US on September 2016.
  • Exondys 51 is approved for a specific subset of DMD patients that are amenable to exon 51 skipping therapies. That accounts for about 13% of DMD patients.
  • In September 2018, the European Medicines Agency (EMA) rejected Sarepta’s application for Exondys 51.
  • The therapy costs about $300,000 US dollar per patient per year.
  • The company is awaiting an FDA decision on its exon 53-skipping therapy, Golodirsen, this summer. It would be appropriate for about 8% of DMD patients.
  • This drug is not currently available in Canada, as Health Canada must approve its use in the Canadian market.