Advances in patient-specific induced pluripotent stem cells shed light on drug discovery for amyotrophic lateral sclerosis

JH Lee, JW Liu, SZ Lin, HJ Harn… - Cell …, 2018 - journals.sagepub.com
… Neurological diseases such as amyotrophic lateral sclerosis (… of new drugs and/or drug
screening for ALS treatment and … a potential therapeutic target in amyotrophic lateral sclerosis. …

[引用][C] A stem cell therapy of amyotrophic lateral sclerosis: From bench to bedside

E Sykova, S Forostyak, P Rychmach, S Konradova… - Cytotherapy, 2018 - Elsevier

[HTML][HTML] Translational study for amyotrophic lateral sclerosis treatment

S Schröder, G Litscher, W Pan - Frontiers in Neurology, 2023 - frontiersin.org
Amyotrophic lateral sclerosis (ALS) is a motor neuron disease characterized by progressive
loss of upper and lower motor neurons. Despite significant scientific efforts in the last …

Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis

TF Gendron, J Chew, JN Stankowski… - … translational medicine, 2017 - science.org
… A limitation in moving such treatments from bench to bedside is a lack of pharmacodynamic
… in gauging biochemical responses to potential treatments. Treating c9ALS patient cells or a …

[HTML][HTML] Inosine in neurodegenerative diseases: From the bench to the bedside

MS Basile, P Bramanti, E Mazzon - Molecules, 2022 - mdpi.com
… its safety and efficacy and its use as a complementary therapy along with other approved
drugs. … Urate levels predict survival in amyotrophic lateral sclerosis: Analysis of the expanded …

[HTML][HTML] Neuroimaging findings in preclinical amyotrophic lateral sclerosis models—How well do they mimic the clinical phenotype? A systematic review

AE Cannon, WE Zürrer, C Zejlon, Z Kulcsar… - Frontiers in Veterinary …, 2023 - frontiersin.org
… This opposes the high attrition of drugs during bench-to-bedside translation … drug development.
These findings emphasize a careful application of these model systems for ALS therapy

Pilot trial of inosine to elevate urate levels in amyotrophic lateral sclerosis

K Nicholson, J Chan, EA Macklin… - Annals of clinical …, 2018 - Wiley Online Library
… 12 weeks of study drug treatment. One participant was … No serious AEs attributed to study
drug and no AEs of special … is rapidly advancing from the bench to the bedside in PD where a …

[HTML][HTML] The novel small molecule TRVA242 stabilizes neuromuscular junction defects in multiple animal models of amyotrophic lateral sclerosis

P Bose, E Tremblay, C Maios, V Narasimhan… - …, 2019 - Springer
… of findings from bench to bedside of potentially effective … a mouse model of amyotrophic
lateral sclerosis. The efficacy of … effects for the treatment of amyotrophic lateral sclerosis. …

Silence superoxide dismutase 1 (SOD1): a promising therapeutic target for amyotrophic lateral sclerosis (ALS)

E Abati, N Bresolin, G Comi, S Corti - Expert opinion on therapeutic …, 2020 - Taylor & Francis
… the small study sample and the low treatment doses received by patients. Another silencing
… RNAi therapy from the bench to the bedside is the lack of an in vivo biomarker of therapeutic

[HTML][HTML] Therapeutic opportunities and challenges of induced pluripotent stem cells-derived motor neurons for treatment of amyotrophic lateral sclerosis and motor …

MK Jaiswal - Neural regeneration research, 2017 - journals.lww.com
drug from the bench to bedside is very expensive, laborious and slow process. The cost of
development and validation of potential therapeutic … with consecutive drug therapies until the …