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.
