WebNusinersen is the first approved drug to treat paediatric and adult patients with SMA. Nusinersen is an antisense oligonucleotide designed to increase the expression of the SMA protein. It was approved by the US Food and Drug Administration in late December 2016 and by the European Medicines Agency in June 2024. WebNusinersen (SPINRAZA™) is a modified antisense oligonucleotide that binds to a specific sequence in the intron, downstream of exon 7 on the pre-messenger …
SPINRAZA prescribing information
Web12 aug. 2024 · Hereditary variant transthyretin amyloidosis (ATTRv) is a rare genetic defect that affects about 5000–10,000 people worldwide, causing amyloidosis secondary to misfolding of mutant transthyretin (TTR) protein fibrils. TTR mutations can cause protein deposits in many extracellular regions of organs, but those deposits in cardiac and … ibm corporation 3039 cornwallis road
Спинална мишићна атрофија — Википедија
Web11 okt. 2012 · This study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with spinal muscular atrophy (SMA). Four dose levels will be evaluated sequentially. Web6 dec. 2016 · Our study shows favourable safety and tolerability, pharmacokinetics, proof-of-concept pharmacodynamics, and a promising clinical response of intrathecal nusinersen. Results informed the design of an ongoing large phase 3, randomised, sham-controlled study of nusinersen in infantile-onset spinal muscular atrophy. Web29 mrt. 2024 · Nusinersen is an antisense oligonucleotide intended for the treatment of spinal muscular atrophy. The pharmacokinetics of nusinersen, following intrathecal … ibm corporate wellness