JNNP: Neonatal-youth data for the treatment of type 1 spinal muscular dystrophy with sodium Nosine - 1 year data from three Asia Pacific regions

Spinal muscular dystrophy type I (SMA1) is the most common and severe SMA.
the results, affected babies were never able to sit alone, with a middle age of 13.5 months.
sodium nosine, an antonymological oligonucleotide that modifies the scissors of living motor neurons (SMN2) to enhance full-length SMN protein expression, is the first of many promising SMA treatments.
clinical trials of SMA1 patients aged 7 months using ENDEAR Nosinasi sodium showed promising movement milestones and improved survival rates.
this cross-border study, this paper provides the first real-world data from Asia on SMA1 patients one year after receiving sodium nosinasi.
This retrospective cohort study, involving eight institutes in three Asian regions (HKSAR, Taiwan and Korea), assessed changes in baseline clinical characteristics, exercise outcomes and breathing requirements of patients involved in SMA1, from pre-treatment (M0) to 6 months (M6) and 10 months after treatment (M10).
from 2017 to 2019, all participants started nosinasitic sodium therapy under the Expanded Access Program (EAP).
baseline data are collected for neonatal screening (if applicable), SMN1 mutations, birth age, sex, age of onset of symptoms, weight, respiratory support, feeding status, musculoskeletal status.
used Hammersmith's Part 2 exercise score (HINE-2), CHOP intent, and athletic performance to record the results of exercise before and after the on-start of sodium in Nosine.
paper uses CHOP ≥ 4 and HINE-2 ≥5 as clinically meaningful improvements.
the demographic data and clinical characteristics of 40 SMA1 patients.
two-thirds of patients have two copies of the SMN2 gene.
more than half of the queue (57 percent) started sodium therapy for nosinasis≤ 2 years old.
9 cases (22.5%) confirmed by neonatal screening, 8 cases began with nosinasitic sodium treatment for 7 months.
all patients started nosinasitic sodium therapy during the symptom period.
20 months (0.35-294 months) of sodium therapy in Nosinas.
36.4 percent (8/22) of patients who started sodium nosinasitic therapy at age ≤2 achieved unassaless sitting and 3 (13.6 percent) achieved assisted standing.
M10, patients with HINE-2 scores ≥5 were 61.1% (11/18), with a median score of 7.5.
, only 6.7 percent (1/15 patients) of patients over 2 years of age who started sodium nossinasitic therapy achieved an unassaless sitting position.
M10, only 7.1 ≥ (1/14) of patients with an HINE-2 score of 5 were patients with a median of 0.5 (Table 1).
despite having a copy of the SMN2 gene, patients who started using nosinasi sodium at 2 months of age received 2-5 points on M10.
patients with three copies of the SMN2 gene had a greater increase in the median HINE-2 score at M6 and M10 than the baseline (p-0.003).
differences were observed in CHOP (p.lt;0.001).
in M10, patients with 3 SMN2 copies (87.5 percent; 7/8) CHOP≥4, while patients with 2 SMN2 copies had only 54.5 percent (6/11).
the only patient who received sodium nosinasim treatment at the age of 2 and be able to sit independently was an adult patient with spinal fusion, with three SMN2 patients receiving nossinol treatment for the first time at the age of 24 and a half.
this multi-country collaborative retrospective observational queue study in Asia provides real data on the first year of treatment, where sodium nosinasi is safe and beneficial for SMA1 patients from newborns to adults.
screening, which promotes early treatment, can maximize the effectiveness of treatment.
Chan SH, Chae J, Chien Y, et al Nusinersen in spinal muscular atrophy type 1 from neonates to young adult: 1-year data from three Asia-Pacific regions Journal of Neurology, Neurosurgery Psython Published Online First: 11 February 2021. doi:10.1136/jnnp-2020-324532MedSci Original Source: MedSci Original Copyright Notice: All notes on this website "Source: Met Medical" or "Source: MedSci Original" text, images and audio and video materials, copyrighted by Mace Medical, not licensed by any media Websites or individuals may not be reproduced, and the authorizing reprints must include "Source: Metz Medicine".
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