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BioWorld - Sunday, July 19, 2026
Home » ALS

Articles Tagged with ''ALS''

CRISPR Cas9 illustration
Neurology/Psychiatric

4DMT, Arbor partner on CRISPR-based therapeutics for CNS diseases

Jan. 4, 2024
4D Molecular Therapeutics Inc. (4DMT) and Arbor Biotechnologies Inc. have established a strategic partnership focused on advancing new AAV-based gene-editing therapies for central nervous system (CNS) diseases with high unmet medical need in both rare and common disease populations.
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Neurology/Psychiatric

Sudo raises series B to advance TYK2 inhibitors for MS, psoriasis

Dec. 21, 2023
Sudo Biosciences Inc. has closed a $116 million series B financing, with the funding raised to be used to advance two investigational TYK2 candidates into the clinic in 2024.
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Illustration demonstrating muscle contraction in amyotrophic lateral sclerosis.
Biomarkers

Increased serum levels of cystatin C are associated with shorter survival in patients with ALS

Dec. 14, 2023
Researchers from West China Hospital of Sichuan University presented data from a study that aimed to investigate the associations between serum cystatin C (CysC) levels and the progression and survival of patients with amyotrophic lateral sclerosis (ALS).
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Scientist, microscope and dropper
Neurology/Psychiatric

Inhibiting C1q reduces ALS-derived damage in mice

Dec. 13, 2023
Amyotrophic lateral sclerosis (ALS) is associated with loss of synaptic connectivity at the neuromuscular junction (NMJ) and loss of motor neurons.
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Neurology/Psychiatric

Merck Sharp & Dohme describes new RIPK1 inhibitors

Dec. 12, 2023
Merck Sharp & Dohme LLC has identified receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis and inflammatory disorders.
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Illustration of motor neuron connecting to muscle fiber
Biomarkers

UCHL1 associated with ALS progression, with impact on survival

Dec. 12, 2023
University of Oxford scientists have presented data from deep proteomics of cerebrospinal fluid (CSF) in search of proteins with diagnostic or prognostic value in amyotrophic lateral sclerosis (ALS). Analysis was performed using CSF samples from 40 ALS patients, 15 controls (healthy individuals) and 8 mimicking conditions.
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Neurology/Psychiatric

HDAC6 inhibitor ACY-738 shows protective effects in axonal degeneration

Dec. 12, 2023
Microtubule acetylation and impaired axonal transport have been proposed as mechanisms causing amyotrophic lateral sclerosis (ALS). Furthermore, histone deacetylase 6 (HDAC6) is known to play a role in acetylation of α-tubulin, a subunit of microtubules. ACY-738 is an HDAC6 inhibitor that has been reported to slow neuron degeneration in Alzheimer’s disease and ALS models by increasing acetylation of α-tubulin.
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Neurology illustration
Neurology/Psychiatric

NX-210c, a promising disease-modifying drug for neurodegenerative disorders

Dec. 11, 2023
Subcommisural organ (SCO)-spondin is a brain-specific glycoprotein essential for neurogenesis with a high impact on neuronal development; thus, reduction in the production of SCO-spondin may lead to a lack of regeneration in neurological disorders.
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Neurology/Psychiatric

Selective P2X7 pathway activation enhances skeletal muscle regeneration in ALS mice

Dec. 11, 2023
Previous research has demonstrated that systemic administration of a P2X7 agonist improved motor performance in a mouse model of amyotrophic lateral sclerosis (ALS) through enhancing satellite cells and the muscle pro-regenerative activity of infiltrating macrophages.
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Neurology/Psychiatric

CAPN2 inhibitor might reduce axonal degeneration in ALS

Dec. 11, 2023
Axonal degeneration is a key early pathogenic driver of amyotrophic lateral sclerosis and other neurodegenerative diseases. Activation of calpain-2 (CAPN2) is thought to be the critical effector behind axonal degeneration. Amylyx Pharmaceuticals Inc. has presented data on their CAPN2 inhibitor antisense oligonucleotide (ASO) AMX-0114 as a potential therapeutic for axonal degeneration.
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