As the survival rate in SMA type 1 increases, the incidence of complications similar to those frequently seen in SMA type 2 and type 3 patients also increases. The follow-up and treatment of patients with SMA should be undertaken by a multidisciplinary team.Turkish...
Baseline demographics and hospital admission data were collected including: the reason for admission, total hospital days, days of critical care, days intubated, discharge diagnosis, doses of nusinersen and treatment complications. Results 11 children (six girls) received nusinersen between May 2017 and...
[1] Dabbous, O., et al., Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1. Adv Ther, 2019. 36(5): p. 1164-1176. [2]Available from: https://www.clinicaltrials.gov/ct...
[1].Servais L, Baranello G, Boespflug-Tanguy O, et al. P.109 FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam in Type 1 spinal muscular atrophy (SMA). Presented at the 27th International Annual Congress of the World Muscle Society, Halifax, Canada, October 11-15, ...
[1] Dabbous, O., et al., Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1. Adv Ther, 2019. 36(5): p. ...
4月28日,渤健公司宣布诺西那生钠注射液正式在中国上市,诺西那生钠注射液是全球首个和目前唯一一个脊髓性肌萎缩症治疗药物。
6. http://media.biogen.com/news-releases/news-release-details/us-fda-approves-biogens-spinrazatm-nusinersen-first-treatment 7. Al-Zaidy S, et al.(2019).Health outcomes in spinal muscular atrophy type 1 following AVXS-101gene replacement therapy. Pediatr Pulmonol.doi:10.1002/ppul.24203. ...
6.Ratni H et al. Discovery of risdiplam, a selective survival motor neuron-2 (SMN2) gene splicing modifier for the treatment of spinal muscular atrophy (SMA). J Med Chem. 2018;61:6501-17. 7.Baranello G et al.FIREFISH Part 1: 1-Year Results on Motor Function in Babies with Type ...
225 type 1 infants born after March 2016 were identified. Their age ranged between 3 months and 8 years, one month. All but 26 had reached two years of age. Only 7 of the 225 patients opted for palliative care and another 4 discontinued treatment after one or a few doses. Another ...
9. Ratni Het al. Discovery of risdiplam, a selective survival motor neuron-2 (SMN2) genesplicing modifier for the treatment of spinal muscular atrophy (SMA). J Med Chem. 2018; 61:6501-17. 10. Roche’s risdiplam showed si...