Journal ArticleDOI
Update of Therapeutic Clinical Trials for Amyotrophic Lateral Sclerosis
Nam-Hee Kim,Min Oh Lee +1 more
- Vol. 17, Iss: 1, pp 1-16
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TLDR
A better understanding of these pathogenic mechanisms and the potential pathological relationship between the various cellular processes have suggested novel therapeutic approaches, including stem cell and genetics-based strategies, providing hope for feasible treatment of ALS.Abstract:
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that is characterized by progressive death of motor neurons in the cortex, brainstem, and spinal cord. Until now, many treatment strategies have been tested in ALS, but so far only Riluzole has shown efficacy of slightly slowing disease progression. The pathophysiological mechanisms underlying ALS are multifactorial, with a complex interaction between genetic factors and molecular pathways. Other motor neuron disease such as spinal muscular atrophy (SMA) and spinobulbar muscular atrophy (SBMA) are also progressive neurodegenerative disease with loss of motor neuron as ALS. This common thread of motor neuron loss has provided a target for the development of therapies for these motor neuron diseases. A better understanding of these pathogenic mechanisms and the potential pathological relationship between the various cellular processes have suggested novel therapeutic approaches, including stem cell and genetics-based strategies, providing hope for feasible treatment of ALS. (Korean J Clin Neurophysiol 2015;17:1-16)read more
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Journal Article
Tirasemtiv Amplifies Skeletal Muscle Response to Nerve Activation in Humans (P4.077)
Fady I. Malik,Richard Hansen,Vipin Vijayakumar,Stoltz Randall,Roger M. Enoka,David J. Morgans,Andrew A. Wolff +6 more
TL;DR: The data confirm that tirasemtiv amplifies the response of skeletal muscle to nerve input in humans, and provides support for further studies of tirasEMtiv as a potential therapy in conditions marked by diminished neuromuscular input.
References
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Journal ArticleDOI
A Controlled Trial of Riluzole in Amyotrophic Lateral Sclerosis
TL;DR: The antiglutamate agent riluzole appears to slow the progression of amyotrophic lateral sclerosis, and it may improve survival in patients with disease of bulbar onset, according to a prospective, double-blind, placebo-controlled trial in 155 outpatients with Amyotrophicateral sclerosis.
Journal ArticleDOI
Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND)
TL;DR: In this article, the authors examined the efficacy of riluzole in prolonging survival, and in delaying the use of surrogates (tracheostomy and mechanical ventilation) to sustain survival.
Reference EntryDOI
Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND).
TL;DR: Riluzole 100 mg daily is reasonably safe and probably prolongs median survival by about two to three months in patients with amyotrophic lateral sclerosis.
Journal ArticleDOI
Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomised trial
Paul H. Gordon,Dan H. Moore,Robert G. Miller,Julaine Florence,Joseph L. Verheijde,Carolyn Doorish,Joan F. Hilton,G Mark Spitalny,Robert B. MacArthur,Hiroshi Mitsumoto,Hans E. Neville,Kevin B. Boylan,Tahseen Mozaffar,Jerry M. Belsh,John Ravits,Richard Bedlack,Michael C. Graves,Leo McCluskey,Richard J. Barohn,Rup Tandan +19 more
TL;DR: The finding that minocycline has a harmful effect on patients with ALS has implications for trials of minocyCline in patients with other neurological disorders, and for how potential neuroprotective agents are screened for use in Patients with ALS.
Journal ArticleDOI
An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: A phase 1, randomised, first-in-man study
Timothy M. Miller,Alan Pestronk,William S. David,Jeffrey D. Rothstein,Ericka Simpson,Stanley H. Appel,Patricia L. Andres,Katy Mahoney,Peggy Allred,Katie Alexander,Lyle W. Ostrow,David A. Schoenfeld,Eric A. Macklin,Daniel A. Norris,Georgios Manousakis,Matthew J. Crisp,Richard Smith,C. Frank Bennett,Kathie M. Bishop,Merit Cudkowicz +19 more
TL;DR: In this paper, the authors evaluated the safety, tolerability, and pharmacokinetics of ISIS 333611 after intrathecal administration in patients with SOD1-related familial amyotrophic lateral sclerosis.