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Tiny Cambridge startup backed by Termeer launches trial of ALS drug

A tiny Cambridge startup backed by the late Henri Termeer has reached an important milestone in its quest to develop a more effective treatment for amyotrophic lateral sclerosis, the debilitating neurological disorder known as ALS. Amylyx Pharmaceuticals, a largely virtual biotech that was founded in 2013 and has just three full-time employees, said Tuesday that it had dosed the first patient in a Phase 2 study of its drug, called AMX0035. Termeer, the founder of Genzyme, was a board member and…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news

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Source: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration - Category: Neurology Authors: Source Type: research
Source: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration - Category: Neurology Authors: Source Type: research
Conclusions: We show that in fibroblasts ofFUS P525L mutation carriers, FUS mislocalized to the cytoplasm where it redistributed into stress granules with likely a dose effect, i.e. a higher number of cells with granules, which persist longer, than in controls and ALS cases. These data represent an early molecular change occurring before ALS onset, suggesting a transient preaggregative state.Neurodegener Dis 2017;17:292-303
Source: Neurodegenerative Diseases - Category: Neurology Source Type: research
Brainstorm Cell Therapeutics (NSDQ:BCLI) said today that the first patients have been enrolled in its Phase III trial evaluating NurOwn as a treatment for amyotrophic lateral sclerosis, or ALS, at Massachusetts General Hospital and UC Irvine Medical Center. The trial, which is slated to enroll 200 patients, is designed to include the pre-specified patient subgroups who saw superior outcomes in the NurOwn Phase II ALS trial. BrainStorm said it expects top-line data from the trial in 2019. Get the full story at our sister site, Drug Delivery Business News. The post BrainStorm enrolls first ALS patient in late-stage NurO...
Source: Mass Device - Category: Medical Devices Authors: Tags: Clinical Trials Neurological Stem Cells Wall Street Beat BrainStorm Cell Therapeutics Inc. Source Type: news
Trial supported by a $16M non-dilutive grant from CIRM Top-line data are expected in 2019 HACKENSACK, N.J. and PETACH TIKVA, Israel, Oct. 16, 2017 -- (Healthcare Sales &Marketing Network) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) today an... Regenerative Medicine, Neurology BrainStorm Cell Therapeutics, NurOwn , stem cell, amyotrophic lateral sclerosis
Source: HSMN NewsFeed - Category: Pharmaceuticals Source Type: news
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
TEL AVIV (Reuters) - BrainStorm Cell Therapeutics said the first patients have been enrolled in a late-stage trial of its treatment for amyotrophic lateral sclerosis (ALS) at Massachusetts General Hospital and UC Irvine Medical Center in California.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Source: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration - Category: Neurology Authors: Source Type: research
We report for the first time, two patients with sporadic limb-onset ALS who developed recurrent acute pancreatitis (AP), with portal vein thrombosis as complication, during treatment with riluzole. We suggest that AP should be considered asa probable rare and severe side effect of treatment with riluzole in patients with ALS. We believe that in patients who develop AP during treatment with riluzole, its withdrawn may prevent recurrent AP and should be discussed with patients.
Source: Journal of Clinical Neuroscience - Category: Neuroscience Source Type: research
Antioxidants&Redox Signaling , Vol. 0, No. 0.
Source: Antioxidants and Redox Signaling - Category: Research Authors: Source Type: research
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