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Home » amyotrophic lateral sclerosis

Articles Tagged with ''amyotrophic lateral sclerosis''

Leqembi launched and PDUFA near, ‘fit-for-growth’ Biogen gets leaner

July 25, 2023
By Randy Osborne
With the launch of intravenous Leqembi (lecanemab-irmb) for Alzheimer’s disease (AD) underway, Priya Singhal, Biogen Inc.’s head of development and interim head of research, said the firm, along with partner Eisai Co. Ltd., aims to “address the long-term duration question” with a subcutaneous version, recently touted at the Alzheimer's Association International Conference.
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Tree in the shape of human head losing leaves
Neurology/Psychiatric

[18F]ACI-19278 TDP-43 ligand shows high target engagement in brain imaging studies

July 20, 2023
Several neurodegenerative disorders have TAR DNA-binding protein 43 (TDP-43) inclusions as a pathological hallmark; thus, the development of PET tracers able to detect TDP-43 aggregates is essential to advance the diagnosis and treatment monitoring in diseases such as frontotemporal dementia, amyotrophic lateral sclerosis and others.
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Illustration demonstrating muscle contraction in amyotrophic lateral sclerosis.
Neurology/Psychiatric

New δ-valerolactone compound demonstrates anti-excitotoxic activity in models of ALS

July 18, 2023
Researchers from Nihon University and Aomori University have discovered a novel δ...
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Drug R&D concept image.
Neurology/Psychiatric

Aeterna Zentaris updates progress of development pipeline

July 14, 2023
Aeterna Zentaris Inc. has offered an update on its development pipeline programs. Macimorelin acetate (AEZS-130), a ghrelin agonist approved and commercialized as a test for adult growth hormone deficiency, is in preclinical development for amyotrophic lateral sclerosis (ALS). The company has now successfully developed an alternative formulation suitable for use in ALS.
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Drug capsule spilling onto brain
Neurology/Psychiatric

Spinogenix advances SPG-302 into clinic in Australia for ALS

July 10, 2023
Spinogenix Inc. is opening enrollment in a first-in-human phase I trial of SPG-302 for the treatment of amyotrophic lateral sclerosis (ALS), having received approval from Australia's Human Research Ethics Committee (HREC).
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Neurology/Psychiatric

Regulus advances preclinical studies of miR-155 inhibitors for ALS

June 21, 2023
Regulus Therapeutics Inc. has provided an update on its preclinical studies evaluating a library of oligonucleotides designed to inhibit miR-155 for the treatment of amyotrophic lateral sclerosis (ALS).
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Qalsody assurance? Biogen ALS biomarkers important but CNM-Au8 data ‘key theme’ as Clene heads to FDA

June 16, 2023
By Randy Osborne
Clene Inc. plans to sit down in the third quarter of this year with the U.S. FDA for talks about approval for gold nanocrystal suspension CNM-Au8, bolstered by favorable biomarker findings in amyotrophic lateral sclerosis (ALS) from the phase II/III Healey ALS platform trial – along with a sizeable time-to-event and survival data package that should drive the conversation.
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Illustration of motor neuron connecting to muscle fiber
Neurology/Psychiatric

Benevolentai advances RARαβ-biased agonist into IND-enabling studies for ALS

June 6, 2023
Benevolentai Ltd. has advanced BEN-34712 into...
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Despite setbacks momentum in ALS builds

May 26, 2023
By Cormac Sheridan
Lack of efficacy brought the development of two investigational agents for amyotrophic lateral sclerosis (ASL) to a halt over the past week. On May 23, Wave Life Sciences Inc. disclosed that its stereopure antisense oligonucleotide WVE-004 failed to demonstrate clinical benefit after 24 weeks of treatment on a phase Ib/IIa trial in familial ALS patients or frontotemporal dementia patients. And on May 25, Apellis Pharmaceuticals Inc. and its partner, Swedish Orphan Biovitrum International AB, said that pegcetacoplan failed to meet its primary endpoint of a one-year phase II trial in patients with sporadic disease.
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PET imaging of RELN-COLBOS (H3447R) carrier brain showing limited aggregation of tau
Neurology/Psychiatric

Brain gene and cell strategies could ‘open the safe’ in neurodegenerative disorders

May 23, 2023
By Mar de Miguel
One of the challenges in designing genetic and cellular strategies is getting the therapy to the right place. This is even more complicated when it comes to the nervous system. The brain is a complex organ that contains the most differentiated and inaccessible cells in human biology. It is an impassable safe, protected by the blood-brain barrier.
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