ITF Pharma Announces FDA Approval of Tiglutik (riluzole) Oral Suspension for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

BERWYN, Pa., Sept. 6, 2018 /PRNewswire/ -- ITF Pharma, a U.S.-based specialty pharmaceutical company and a subsidiary of Italfarmaco, a privately-held European specialty pharmaceutical company, committed to investing in and commercializing impactful...
Source: Drugs.com - New Drug Approvals - Category: Drugs & Pharmacology Source Type: news

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In recent years researchers have investigated changes in alternative splicing in the context of aging and age-related disease. It is thought to be important in cellular senescence, for example, but that is just one line item in the bigger picture. A given gene can code for multiple different proteins, and alternative splicing is the name given to the processes by which those different proteins are produced. A gene contains discrete DNA sequences called exons and introns, the former passed into the protein production process, and the latter removed during RNA splicing. The canonical protein produced from this genetic bluepr...
Source: Fight Aging! - Category: Research Authors: Tags: Medicine, Biotech, Research Source Type: blogs
Publication date: Available online 21 March 2019Source: Journal of Molecular BiologyAuthor(s): Sei-Kyoung Park, Sangeun Park, Susan W. LiebmanAbstractThe trans-activating response DNA-binding protein 43 (TDP-43) is a transcriptional repressor and splicing factor. TDP-43 is normally mostly in the nucleus, although it shuttles to the cytoplasm. Mutations in TDP-43 are one cause of familial amyotrophic lateral sclerosis (ALS). In neurons of these patients, TDP-43 forms cytoplasmic aggregates. In addition, wild-type TDP-43 is also frequently found in neuronal cytoplasmic aggregates in patients with neurodegenerative diseases n...
Source: Journal of Molecular Biology - Category: Molecular Biology Source Type: research
The aim of our study was to evaluate the role of mutations in theMAPT gene in patients with pure amyotrophic lateral sclerosis (ALS). A cohort of 120 ALS patients, both sporadic and familial, without cognitive impairment was analyzed by next-generation sequencing with a multiple-gene panel comprising 23 genes, includingMAPT, known to be associated with ALS and frontotemporal dementia. The presence of theC9orf72 expansion was also investigated. Twelve patients had mutations in theSOD1, TARDBP, MATR3, andFUS genes, while 10 patients carried theC9orf72 expansion. One female patient was found to carry the D348G mutation inMAPT...
Source: Neurodegenerative Diseases - Category: Neurology Source Type: research
Stress granules (SGs) are non-membrane-bound RNA-protein granules that assemble through phase separation in response to cellular stress. Disturbances in SG dynamics have been implicated as a primary driver of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), suggesting the hypothesis that these diseases reflect an underlying disturbance in the dynamics and material properties of SGs. However, this concept has remained largely untestable in available models of SG assembly, which require the confounding variable of exogenous stressors. Here we introduce a light-induc...
Source: eLife - Category: Biomedical Science Tags: Cell Biology Source Type: research
Abstract The aim of our study was to evaluate the role of mutations in the MAPT gene in patients with pure amyotrophic lateral sclerosis (ALS). A cohort of 120 ALS patients, both sporadic and familial, without cognitive impairment was analyzed by next-generation sequencing with a multiple-gene panel comprising 23 genes, including MAPT, known to be associated with ALS and frontotemporal dementia. The presence of the C9orf72 expansion was also investigated. Twelve patients had mutations in the SOD1, TARDBP, MATR3, and FUS genes, while 10 patients carried the C9orf72 expansion. One female patient was found to carry t...
Source: Neuro-Degenerative Diseases - Category: Neurology Authors: Tags: Neurodegener Dis Source Type: research
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Source: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration - Category: Neurology Authors: Source Type: research
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Source: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration - Category: Neurology Authors: Source Type: research
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Source: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration - Category: Neurology Authors: Source Type: research
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Source: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration - Category: Neurology Authors: Source Type: research
Bridging biophysics and neurology: aberrant phase transitions in neurodegenerative disease, Published online: 19 March 2019; doi:10.1038/s41582-019-0157-5In this Review, Nedelsky and Taylor review the evidence that disturbances in phase transition dynamics and the material properties of ribonucleoprotein granules underlie the pathogenesis of many neurodegenerative diseases, including forms of amyotrophic lateral sclerosis and frontotemporal dementia, among others.
Source: Nature Reviews Neurology - Category: Neurology Authors: Source Type: research
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