URGENT DEADLINE: Please complete AS SOON AS POSSIBLE or before 7 July 2017. 

This survey is being conducted on behalf of Cure SMA Canada in collaboration with the Canadian Organization for Rare Disorders (CORD).  Your feedback is URGENTLY needed if you are a caregiver for or a person with spinal muscular atrophy (SMA) of any type.  Canadian patients have the opportunity to provide input to the Canadian Agency for Drugs and Technologies in Health (CADTH) and specifically the Common Drug Review (CDR), the agency that will recommend (or not) whether Spinraza (nusinersen) will be funded for SMA patients.

You do not need to have experience with SPINRAZA to respond to this survey.  It should take only about 15 minutes.

Your responses will NOT be personally identified.  All responses will be summarized as part of the submission to CDR.  In our experience, the CDR committee listens very carefully to patient input.  Patient input is very important to assuring the committee makes the right decision.

Thank you in advance for your help.

* 1. Spinal muscular atrophy is typically classified into four types.

SMA Type 1 is the most severe form and usually diagnosed between 3 - 6 months of age.  A child with type 1 is typically never able to lift his/her head or accomplish the normal gross and fine motor skills expected early on in infancy.  Swallowing may be difficult and the child may have not be able to breathe deeply enough to get sufficient oxygen in and carbon dioxide out.

SMA Type II is usually diagnosed before 2 years of age.  The child may sit unsupported but is often unable to come to a sitting position without assistance.  Swallowing is usually not a problem but the child may have difficulty taking deep breaths while sleeping.

SMA Type III, or Juvenile Muscular Atrophy, varies in age of onset but is typically diagnosed by age 3.  The can stand alone and walk but may fall more frequently and, over time, have difficulty in getting up from the floor, and may be unable to run. Feeding or swallowing difficulties in childhood are very uncommon.

SMA Type IV, Adult Onset SMA, is typically diagnosed after age 35.  It is characterized by onset of weakness, and persons affected lose function over time.  Swallowing and respiratory function are rarely affected.

Which of the following BEST describes the diagnosis of SMA for you (or the person you care for).

* 2. Please describe how SMA has affected you and your family.  What has been the impact on the person affected?  How has SMA affected the family and others outside of the immediate family?  What has been the impact socially, financially, and psychologically?

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