Inactivation of ATG7, one of the genes that controls autophagy — a process in which cells degrade or recycle components that are damaged or no longer needed — is linked to the onset of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), a study says. Results from…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Apic Bio‘s APB-102, an investigational gene therapy aiming to treat familial amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene. “This orphan drug designation represents an important…
The Chinese caterpillar fungus (Hirsutella sinensis), used in traditional Chinese medicine, minimized motor neuron loss and extended the lifespan in a mouse model of amyotrophic lateral sclerosis (ALS), a study has found. The study, “Therapeutic effects of hirsutella sinensis on the disease onset and progression of amyotrophic lateral…
Scientists who dissected the structure, and mechanism of action, of the protein Cdc48 — dubbed the “Swiss Army knife of cells” — say it can be explored as a therapeutic target for several disorders, including amyotrophic lateral sclerosis (ALS). The findings of the study, “Structure of the…
Posture impairments in amyotrophic lateral sclerosis (ALS) are linked to deficits in trunk control and in the mechanisms that control the body’s response to surface tilts. Moreover, these abnormalities also correlated to disease severity. Those findings are in the study “Abnormal trunk control determines postural abnormalities in Amyotrophic…
Caregivers of people with amyotrophic lateral sclerosis (ALS) and progressive muscular atrophy (PMA) say a Dutch support program — one based on Acceptance and Commitment Therapy — empowered them to make better choices in line with their own needs, and to cope with distress, a study shows. The…
Histamine-related genes are promising disease biomarkers and therapeutic targets to slow amyotrophic lateral sclerosis (ALS) progression, a mouse study suggests. The study, “Histaminergic transmission slows progression of amyotrophic lateral sclerosis,” was published in the Journal of Cachexia, Sarcopenia and Muscle. ALS is a progressive neurodegenerative disorder…
Oxford BioDynamics has joined the REFINE-ALS study, a project designed to detect and measure the levels of specific biomarkers among people with amyotrophic lateral sclerosis (ALS), Mitsubishi Tanabe Pharma America (MTPA) announced. REFINE-ALS is sponsored by the MTPA and led by the Massachusetts General Hospital (MGH)…
NPT520-34, an investigational small molecule for the treatment of Parkinson’s disease and amyotrophic lateral sclerosis (ALS), is being tested on healthy individuals in a new Phase 1 clinical trial, its developer Neuropore Therapies has announced. Brain inflammation is a common feature among many neurodegenerative disorders, including Parkinson’s,…
Reldesemtiv (formerly known as CK-2127107) reduces lung function decline and overall disease progression, without significant adverse events, in people with amyotrophic lateral sclerosis (ALS), a Phase 2 trial shows. The study, however, did not meet its primary endpoint. The…
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