Treatments for Spinal Muscular Atrophy (SMA)

While there is no cure for SMA, there are three treatments that have been approved by Health Canada.

Evrysdi (generic name: risdiplam)

What is it?
EVRYSDI is a pre-mRNA splicing modifier of survival of motor neuron 2 (SMN2).

How does it work?
EVRYSDI works by helping the body make more of a protein called “SMN protein”. EVRYSDI increases the amount of SMN protein in the body, which helps to treat SMA.

 

Age range for treatment
EVRYSDI is for use in children 2 months of age and older and in adults with SMA

Spinraza (generic name: nusinersen)

What is it?
SPINRAZA belongs to a group of medicines called anti-sense oligonucleotides (ASO).

 

How does it work?
SPINRAZA works by helping the body to produce more of the SMN protein that people with SMA need. This may reduce the loss of nerve cells and improve muscle strength.

 

Age range for treatment
SPINRAZA may be given to children, adolescents or adults with SMA.

There is limited experience of SPINRAZA in people over 18 years of age.

There is no experience of SPINRAZA in people over 65 years of age.

Zolgensma (generic name: onasemnogene abeparvovec)

What is it?
Zolgensma is a type of medicine called ‘gene therapy’. It contains the active ingredient onasemnogene abeparvovec, which contains human genetic material.

 

How does it work?
Zolgensma supplies a dully functioning copy of the survival motor neuron 1 (SMN1) gene, which helps cells produce SMN protein necessary for the survival of nerves that control muscles (motor neurons). Patients treated with ZOlgensma showed improvement relative to the usual progress of SMA.

 

Age range for treatment
Zolgensma is used to treat babies and young children with SMA.

Coverage of Treatments in Canada

In Canada, each province’s coverage is based on specific criteria for pediatric use while adult access is decided on a case-by-case basis except in Quebec where all SMA patients are eligible based on criteria. Please discuss your eligibility with your health-care provider.

 

Click on each province below for more detailed information about coverage in each province

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Coverage by Province