Saskatchewan treatments

ROCHE: Evrysdi – Risdiplam (pg.67


Effective March 1, 2022 – Available through the Exceptional Drug Status Program; criteria are not public

BIOGEN: Nusinersen – Spinraza


Initiation Criteria
1. Pre-symptomatic patients with two or three copies of the SMN2 gene, OR

2. Had the disease for <6 months, two copies of SMN2, and symptom onset after the first week after birth and on, or before seven months of age; OR 3. Patients under the age of 18 with symptom onset > 6 months of age, and never achieved the ability to walk independently.†

Stopping Criteria
1. For those pre-symptomatic at initiation: no improvement on HINE-2, CHOP INTEND, or HFMSE, OR

2. For those symptomatic at initiation: no improvement in HINE-2, CHOP INTENT, or HFMSE, OR

3. Permanent invasive ventilation required

“…type II and type III patients over the age of 18 are encouraged to talk to their treating physician to apply for a case by case coverage.”

NOVARTIS: Onasemnogene abeparvovec – Zolgensma


Exception Drug Status (EDS)


Cure SMA Canada Conference August 19 – 21, 20220 Montreal, Quebec Le Centre Sheraton Montréal 1201 Blvd Rene-Levesque West

‘This disease will slowly rob me of everything’: Why are Canadians living with rare diseases waiting for treatment?

COVID has proven we can quickly set up infrastructure without long-term clinical data. It’s time to do the same with rare diseases, says Durhane Wong-Rieger, president and CEO of Canadian Organization for Rare Disorders. Vanessa Hrvatin, Feb 28 2022 Sasha Kirkland, pictured with her husband, Mark and son Maks, lives with spinal muscular atrophy (SMA).…

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