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Pathogenic variants have additive effect on ALS survival

Genetic variants that modify survival in amyotrophic lateral sclerosis (ALS) can act on their own or in unison, having an additive effect and significantly worsening a patient’s outcome, researchers report in the journal Neurology.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.

α-synuclein seed amplification potential for early Parkinson’s disease diagnosis

An assay for identifying the build-up of the misfolded α-synuclein protein in cerebrospinal fluid could enhance the detection of Parkinson’s disease in the early stages before the onset of motor symptoms, suggests research.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.

Promising results for focused ultrasound ablation in Parkinson’s disease

Focused ultrasound ablation of the globus pallidus internus improved symptoms in a substantial proportion of people with Parkinson’s disease in a randomised trial, but it comes with side effects.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.

Study sheds light on genetic cause of late-onset cerebellar ataxia

A dominantly inherited, intronic repeat expansion in the fibroblast growth factor 14 gene is associated with late-onset cerebellar ataxia, shows research published in The New England Journal of Medicine.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.

Promising data for lecanemab in early Alzheimer’s disease

Treatment with the monoclonal antibody lecanemab is associated with less decline in measures of cognition and function as well as a reduction in amyloid burden relative to placebo in people with early Alzheimer’s disease, suggest phase 3 trial data.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.

FIREFISH 2-year data show continued benefit of risdiplam for type 1 SMA

The 24-month results from part 2 of the open-label FIREFISH trial suggest that prolonged treatment with risdiplam is associated with continued improvements in motor function and development in infants with type 1 spinal muscular atrophy.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.

VALOR hints at potential benefit of tofersen for SOD1 amyotrophic lateral sclerosis

Data from the open-label extension of the VALOR trial suggest that treatment with the intrathecally administered antisense oligonucleotide tofersen may modify the disease course of amyotrophic lateral sclerosis due to SOD1 mutations and lead to improvements in function.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.

Monoclonal antibodies disappoint in early Parkinson’s disease

Two monoclonal antibodies targeting aggregated α-synuclein have failed to improve clinical or imaging measures of Parkinson’s disease progression in the early-stage setting.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.

Ultrahigh-dose methylcobalamin shows promise for early-stage ALS

Treatment with ultrahigh-dose methylcobalamin may slow the functional decline of amyotrophic lateral sclerosis patients with early-stage disease and a moderate progression rate, indicate Japanese trial data published in JAMA Neurology.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.