Amyotrophic lateral sclerosis & frontotemporal degeneration
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Amyotroph Lateral Scler Frontotemporal Degener · Mar 2014
ReviewPredicting success: optimizing phase II ALS trials for the transition to phase III.
Amyotrophic lateral sclerosis (ALS) research is advancing quickly, but the transition from phase II to phase III trials remains particularly challenging. In part, this is because of the paradox of phase II ALS trials - they are expected to inform researchers about safety, tolerability, dosage selection, and efficacy using a small number of patients, and relying on essentially the same outcome measures used in phase III trials. We examined pharmacokinetics in the cerebrospinal fluid and pharmacodynamic markers to demonstrate target engagement. ⋯ Primary endpoints should be pre-specified. Inclusion criteria should be used to reduce heterogeneity and target a relevant subpopulation of people with ALS when possible. Multiple phase II trials might be required before moving to a large phase III trial.
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Amyotroph Lateral Scler Frontotemporal Degener · Mar 2014
Emotion processing deficits distinguish pure amyotrophic lateral sclerosis from frontotemporal dementia.
Amyotrophic lateral sclerosis (ALS) is a multisystem disease that overlaps with frontotemporal dementia (FTD). Although FTD patients exhibit prominent deficits in emotion perception and social cognition, these domains have received relatively little attention in ALS. Moreover, direct comparisons between ALS and FTD on emotion processing tasks remain lacking. ⋯ We conclude that patients with FTD-ALS and FTD show similar, significant impairments in emotional processing. By contrast, ALS patients without dementia exhibit preserved emotion processing. Performance on emotion processing tasks may provide a useful clinical tool in identifying those with early FTD-ALS.
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Amyotroph Lateral Scler Frontotemporal Degener · Dec 2013
ReviewSafety and administration of treatment with botulinum neurotoxin for sialorrhoea in ALS patients: review of the literature and a proposal for tailored treatment.
Botulinum neurotoxin (BoNT) is a second-line treatment of sialorrhoea in ALS (amyotrophic lateral sclerosis) patients. This article is a review of the published literature concerning safety and administration of this treatment to ALS patients. A PubMed search was performed. ⋯ In conclusion, BoNT treatment for sialorrhoea in ALS patients is safe with few adverse effects. The authors advocate for the implementation of a personalized treatment strategy. Special precautions must be taken when patients do not have the assistance of a ventilator and a feeding tube.