1 option
Clinical review report : (Novartis Pharmaceuticals Canada Inc.) : indication: for the treatment of pediatric patients with 5q spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and: 3 or fewer copies of SMN2 gene; or infantile-onset SMA. Onasemnogene abeparvovec (Zolgensma) / Canadian Agency for Drugs and Technologies in Health.
- Format:
- Book
- Author/Creator:
- Canadian Agency for Drugs and Technologies in Health, author.
- Language:
- English
- Subjects (All):
- Outcome assessment (Medical care).
- Vision disorders--Treatment.
- Vision disorders.
- Physical Description:
- 1 online resource (108 pages) : illustrations
- Edition:
- Version: final (with redactions).
- Place of Publication:
- Canada : Canadian Agency for Drugs and Technologies in Health, 2021.
- Summary:
- The objective of this report was to perform a systematic review of the beneficial and harmful effects of voretigene neparvovec administered as a single dose of 1.5 × 1011 vector genomes in each eye via subretinal injection for the treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
- Notes:
- Description based on publisher supplied metadata and other sources.
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