Evrysdi – Risdiplam

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Disponible:

À compter du 16 février 2022 : traitement offert dans le cadre du programme Exceptional Expensive Drugs for Rare Disease Process (aucun critère imposé)

Spinraza – Nusinersen

https://archive.news.gov.bc.ca/releases/news_releases_2017-2021/2019HLTH0164-002427.pdf

Available:

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

British Columbia covers only pediatric patients up to the age of 12.