[1] Oki R, Izumi Y, Fujita K, et al. Efficacy and Safety of Ultrahigh-Dose Methylcobalamin in Early-Stage Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial. JAMA Neurol. 2022;79(6):575-583. [2] Elia AE, Lalli S, Monsurrò...
Treatment effects by UNC13A genotypes were evaluated in a randomized, placebo-controlled, Phase 3 clinical trial in ALS participants (Cudkowicz et al. 2022). Clinical response at Week-28 using the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), as well as neurofilament ...
神经炎症:虽然ALS主要不是由炎症引起,但研究发现中枢神经系统内的小胶质细胞和星形胶质细胞被过度激活,释放炎性因子加剧神经元损伤 (Positive Results for Phase 2/3 Masitinib Clinical Trial Announced | The ALS Association)。例如ALS患者脑脊液中发现了炎症标志物升高。一些新药正尝试通过抑制神经系统的炎症来延缓疾病...
2022 Relyvrio (AMX0035) Amylyx Pharmaceuticals Removed from the market on 4/4/2024 due to clinical trial results 2023 Qalsody® (tofersen) Biogen Slows the rate of ALS progression for the approximately 2% of ALS patients who have an SOD1 gene mutationClinical...
Paganoni S, Hendrix S, Dickson SP, et alEffect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trialJournal of Neurology, Neurosurgery & P...
Results: We examined clinical actions of gait, step-up/down, arm rotation, eating action, writing action, swallowing and ankle range of motion using 3D motion capture as actions in ALSFRS- R score that is current clinical scales for ALS patient. We could develop novel clinical algorithms to ...
recent failures in clinical trial of two antisense oligonucleotides (ASOs) targetingC9orf72sense repeat-containing transcripts (BIIB078; clinicaltrials.gov: NCT03626012 and WVE-004; clinicaltrials.gov: NCT04931862), despite showing target engagement and lowering of the sense transcript, highlight the ...
was evaluated in a Phase 3 clinical trial, and this formulation received FDA approval on May 12, 2022. Approval of this formulation will allow the drug to be taken easily at home, orally or via feeding tube, rather than requiring an IV. Research advances and the promise of targeted ...
[4] Berry JD, Cudkowicz ME, Windebank AJ, Staff NP, Owegi M, Nicholson K, McKenna-Yasek D, Levy YS, Abramov N, Kaspi H, Mehra M, Aricha R, Gothelf Y, Brown RH. NurOwn, phase 2, randomized, clinical trial in patients with ALS: Safety, clinical, and biomarker results. Neurology...
“The best thing for a clinical trial is for it to be short and decisive — to get the effective drugs to patients and the ineffective drugs out of the way to not take away resources. Natural history data and disease progression biomarkers are the drivers of that,” he said. PRO-ACT, ...