Berwyn pharma firm gets approval for ALS drug

The Food and Drug Administration has approved ITF Pharma ’s new drug application for Tiglutik, a treatment for amyotrophic lateral sclerosis or ALS. Tiglutik is, according to ITF Pharma, the first and only easy-to-swallow thickened riluzole liquid for ALS. The medicine is administered twice daily by oral syringe. ALS, also known as Lou Gehrig’s dise ase, is a progressive and ultimately fatal neurodegenerative disease marked by a gradual degeneration of nerve cells of the central nervous system…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news

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A clinically useful model to prognose onset of respiratory insufficiency in amyotrophic lateral sclerosis (ALS) would inform disease interventions, communication and clinical trial design. We aimed to derive and validate a clinical prognostic model for respiratory insufficiency within 6 months of presentation to an outpatient ALS clinic. We used multivariable logistic regression and internal cross-validation to derive a clinical prognostic model using a single-centre cohort of 765 ALS patients who presented between 2006 and 2015. External validation was performed using the multicentre Pooled Resource Open-Access ALS C...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Respiratory clinical practice Original Articles: Respiratory clinical practice Source Type: research
In the 1980s the United States Marine Corps had an advertising campaign built around the phrase, "The Marines are looking for a few good men" (figure 1). This non-gender-neutral slogan would probably not succeed today, but the sentiment could be applied toward the care of amyotrophic lateral sclerosis (ALS). We're looking for a few good pulmonologists. ALS is a progressive neuromuscular disease in which there is degeneration of both upper and lower motor neurons, leading to diffuse muscle weakness and spasticity. It is commonly known as Lou Gehrig's disease in the USA, and motor neurone disease in the UK. As ALS ...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Editorials Source Type: research
Qi Li*, Zhaohua Lu, Ning Gao and Jingjing Yang School of Computer Science and Technology, Changchun University of Science and Technology, Changchun, China Objective: P300-speller is the most commonly used brain-computer interface (BCI) for providing a means of communication to patients with amyotrophic lateral sclerosis. However, the performance of the P300-speller BCI is still inadequate. We investigated whether the performance of P300-speller can be further improved by increasing the mental effort required of the user. Methods: We designed two active mental tasks for a P300-speller based on a differently colo...
Source: Frontiers in Human Neuroscience - Category: Neuroscience Source Type: research
In this study, we used a monosynaptic rabies tracing technique to label the whole-brain inputs to specific cell types in the MOp and MOs simultaneously in a same transgenic mouse. First, 150 nl AAV helper mixtures were injected into the ipsilateral MOp (AP:1.54 mm, ML:1.70 mm, DV:-1.50 mm) and MOs (AP:1.54 mm, ML:0.50 mm, DV:-1.35 mm) in Thy1-cre or Vgat-cre mice respectively, mixed with rAAV2/9-Ef1α-DIO-BFP-2a-TVA-WPRE-pA and rAAV2/9-Ef1α-DIO-RG-WPRE-pA as the ratio of 1:2. Three weeks later, 300 nl RV-ΔG-EnVA-EGFP and RV-ΔG-EnVA-Dsred were injected into the two subregions of the MC respectively. O...
Source: Frontiers in Neuroanatomy - Category: Neurology Source Type: research
The hexanucleotide repeat expansion in the C9orf72 gene is the most common genetic variant found in individuals with sporadic amyotrophic lateral sclerosis (ALS), occurring at a frequency of between 7 and 11% in cohorts of European ancestry. While limited data suggest that C9-expansions (>30 repeats) are less frequent in African-Americans with ALS, there is no data on the frequency of C9-expansions among ALS subjects residing in Africa. We therefore investigated the frequency of this expansion mutation (using repeat-primed PCR) in a cohort of 143 South Africans (SA) with ALS.
Source: Journal of the Neurological Sciences - Category: Neurology Authors: Source Type: research
AbstractThe pressure to develop pharmacological therapeutic interventions in the field of the rapidly progressing, fatal disease amyotrophic lateral sclerosis (ALS) is traditionally high. Cannabinoids have been discussed for decades as potential neuroprotective agents for ALS because of their antiexcitatory, anti ‐inflammatory, antiapoptotic and anticatabolic properties. This Editorial highlights a study by Urbi et al in the current issue of the Journal of Neurochemistry, in which the authors performed a Systematic Review and come to the conclusion that cannabinoids seem to have a small, but consistent eff ect on surviva...
Source: Journal of Neurochemistry - Category: Neuroscience Authors: Tags: Editorial Highlight Source Type: research
AbstractAquaporin 4 (AQP4) is a primary water channel found on astrocytes in the central nervous system (CNS). Besides its function in water and ion homeostasis, AQP4 has also been documented to be involved in a myriad of acute and chronic cerebral pathologies, including autoimmune neurodegenerative diseases. AQP4 has been postulated to be associated with the incidence of a progressive neurodegenerative disorder known as amyotrophic lateral sclerosis (ALS), a disease that targets the motor neurons, causing muscle weakness and eventually paralysis. Raised AQP4 levels were noted in association with vessels surrounded with sw...
Source: Neurological Sciences - Category: Neurology Source Type: research
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Source: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration - Category: Neurology Authors: Source Type: research
Discussion We have found that, in the human genome, the promoter regions of ID-associated genes are uniquely enriched in MER41 LTRs. More specifically, nine ID-associated genes that are putatively important in cognitive evolution exhibit MER41 LTRs in their promoter regions. As more than 100 families of HERV are integrated into our genome, it was important to determine whether our findings are specific to MER41 and to ID-associated genes, and if so to what extent. Among the 133 families of HERV explored here, MER41 is the only family whose LTRs were found with statistically high frequency in the promoter regions of ID-ass...
Source: Frontiers in Genetics - Category: Genetics & Stem Cells Source Type: research
Conclusions: We demonstrated in the SOD1G93A model of ALS that increased levels of several cytokines were associated with a shorter lifespan. However, their role as prognostic biomarkers is unclear as their expression was very variable depending on both the disease stage and the subject. Nevertheless, cytokines may be potential therapeutic targets. Introduction Amyotrophic Lateral Sclerosis (ALS) is one of the most common rare diseases of unknown origin that leads to progressive motor neuron degeneration and muscle denervation (1). In particular, it has been described that either distal axonopathy or neuromuscular ju...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
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