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Flex Pharma begins Phase II trial of FLX-787 for ALS in US

Flex Pharma has begun a Phase II clinical trial (COMMEND) of FLX-787 in the US to treat patients suffering from motor neuron disease (MND) with a focus on amyotrophic lateral sclerosis (ALS).
Source: Drug Development Technology - Category: Pharmaceuticals Source Type: news

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Source: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration - Category: Neurology Authors: Source Type: research
Developments in eye-gaze technology – which converts minute movements of the eye into spoken words – are opening up undreamed of opportunities for people with motor neurone syndromeSteve Thomas and I are talking about brain implants. Bonnie Tyler ’s Holding Out For a Hero is playing in the background and for a moment I almost forget that a disease has robbed Steve of his speech. The conversation breaks briefly; now I see his wheelchair, his ventilator, his hospital bed.Steve, a software engineer, was diagnosed with ALS (amyotrophic lateral sclerosis, a type ofmotor neurone disease) aged 50. He knew i...
Source: Guardian Unlimited Science - Category: Science Authors: Tags: Motor neurone disease Medical research Technology Society Science Health & wellbeing Life and style Source Type: news
Frontotemporal dementia (FTD), the second most common form of dementia in people under 65 years of age, is characterized by progressive atrophy of the frontal and/or temporal lobes. FTD overlaps extensively with the motor neuron disease amyotrophic lateral sclerosis (ALS), especially at the genetic level. Both FTD and ALS can be caused by many mutations in the same set of genes; the most prevalent of these mutations is a GGGGCC repeat expansion in the first intron of C9ORF72. As shown by recent intensive studies, some key cellular pathways are dysregulated in the ALS-FTD spectrum disorder, including autophagy, nucleoc...
Source: EMBO Journal - Category: Molecular Biology Authors: Tags: Molecular Biology of Disease, Neuroscience Review Source Type: research
While the death of motor neuron is a pathological hallmark of amyotrophic lateral sclerosis (ALS), defects in other cell types or organs may also actively contribute to ALS disease progression. ALS patients experience progressive skeletal muscle wasting that may not only exacerbate neuronal degeneration, but likely has a significant impact on bone function. In our previous published study, we have discovered severe bone loss in an ALS mouse model with overexpression of ALS-associated mutation SOD1G93A (G93A).
Source: Bone - Category: Orthopaedics Authors: Tags: Full Length Article Source Type: research
AbstractThe mutation of vesicle-associated membrane protein-associated protein B (VAPB) was proved to cause family amyotrophic lateral sclerosis (FALS). Only two mutations ofVAPB associated with ALS have been reported (p.Pro56Ser and p.Thr46Ile). Here we reported a Chinese Han FALS family caused by a novelVAPB point mutation. The clinical materials of one Chinese Han FALS family were collected. The genetic analysis was carried out by target sequencing and further verified by Sanger sequencing. One novel mutation of c.167C>A (p.Pro56His) onVAPB was found in the proband. The age at onset of the proband was 48 with the ons...
Source: Journal of Neurology - Category: Neurology Source Type: research
CONCLUSIONS: Moderate-quality evidence from a single RCT of NIV in 41 participants suggests that it significantly prolongs survival, and low-quality evidence indicates that it improves or maintains quality of life in people with ALS. Survival and quality of life were significantly improved in the subgroup of people with better bulbar function, but not in those with severe bulbar impairment. Adverse effects related to NIV should be systematically reported, as at present there is little information on this subject. More RCT evidence to support the use of NIV in ALS will be difficult to generate, as not offering NIV to the co...
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
Amyotrophic lateral sclerosis (ALS), a terminal degenerative disease of the upper and lower motor neurons (LMN), lacks reliable lower motor neuron disease progression biomarker. Clinical assessment, functional scales, and routine electrophysiological parameters are insensitive in detecting subtle lower motor neuron degeneration as muscle strength and compound muscle action potential (CMAP) can remain stable despite progressive subclinical LMN loss and reinnervation (Neuwirth et al., 2017).
Source: Clinical Neurophysiology - Category: Neuroscience Authors: Source Type: research
Abstract Macroautophagy/autophagy is the main intracellular catabolic pathway in neurons that eliminates misfolded proteins, aggregates and damaged organelles associated with ageing and neurodegeneration. Autophagy is regulated by both MTOR-dependent and -independent pathways. There is increasing evidence that autophagy is compromised in neurodegenerative disorders, which may contribute to cytoplasmic sequestration of aggregation-prone and toxic proteins in neurons. Genetic or pharmacological modulation of autophagy to promote clearance of misfolded proteins may be a promising therapeutic avenue for these disorder...
Source: Autophagy - Category: Cytology Authors: Tags: Autophagy Source Type: research
Publication date: 2017 Source:Handbook of Clinical Neurology, Volume 145 Author(s): Tibor Hortobágyi, Nigel J. Cairns Amyotrophic lateral sclerosis (ALS) is the major motor neuron disorder. The hallmark features are progressive, irreversible motor neuron loss leading to denervation atrophy of muscles and death, usually within 5 years of disease onset. The hallmark proteins of the pathognomonic inclusions are SOD-1, TDP-43, or FUS; rarely the disease is caused by mutation of the respective genes. Frontotemporal lobar degeneration (FTLD) is genetically, neuropathologically, and clinically heterogeneous and may presen...
Source: Handbook of Clinical Neurology - Category: Neurology Source Type: research
Though we do not yet have a cure for amyotrophic lateral sclerosis (ALS), we can provide treatment, and the host of medical and other interventions provided by ALS specialists and multidisciplinary care teams increases survival and substantially improves quality of life for patients and their families. Dysphagia is one of the most consequential symptoms in ALS, and ultimately affects the majority of patients. It causes dehydration, weight loss, choking, and chronic aspiration, which substantially increase the risk of potentially fatal aspiration pneumonia. Weight loss alone worsens progression and survival in ALS,1 pr...
Source: Neurology - Category: Neurology Authors: Tags: EDITORIALS Source Type: research
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