Outside-In vs Inside-Out Paradigms in Patients with Multiple Sclerosis
Study researchers examined the different paradigms on the pathophysiology of MS, the outside-in and the inside-out theories.
Neurodegenerative Diseases
FDA: More Data Needed to Support Arimoclomol in Niemann-Pick Disease Type C
Arimoclomol is an investigational oral drug candidate that is designed to amplify and sustain the cellular production of heat shock proteins.
Researchers to Study Optimized Home-Based Respiratory Management in ALS
Researchers from South America presented a protocol for a randomized trial to study the effects of an optimized home-based respiratory care approach in patients with ALS.
Gene Therapy Candidate Fast Tracked for GM1 Gangliosidosis
LYS-GM101 is a gene therapy designed to replace the gene mutation and restore the production of the beta-galactosidase enzyme.
Long-Term Low-Dose Sargramostim Therapy Restores Immune Homeostasis in Parkinson Disease
Because sargramostim therapeutic use has been offset by dose-dependent adverse events, the researchers performed a reduced drug dose regimen to evaluate safety and to discover novel disease-linked biomarkers during sargramostim treatments for 1 year.
Lifestyle and Genetics Interact During Presymptomatic Stage of ALS
Investigators aimed to assess lifestyle during the presymptomatic phase of ALS, stratified by C9orf72 mutation, and examine evidence about whether lifestyle factors are causative factors.
Cortical Thinning in Alzheimer Disease Tied to Neighborhood-Level Disadvantage
Researchers compared levels of cortical thinning and cognitive decline of patients with Alzheimer disease based on their neighborhood of residence’s level of disadvantage.
Tilavonemab Not Effective in Progressive Supranuclear Palsy
Researchers assessed the safety and efficacy of tilavonemab for treating progressive supranuclear palsy, a neurodegenerative disorder linked to tau protein aggregation.
Subtype of Parkinson Disease Correlated With Local Gyrification Index
Researchers sought to identify cortical gyrification and white matter microstructural changes in patients with Parkinson disease with akinetic-rigid and tremor-dominant subtypes.
Gene Therapy: A Promising Future for Inherited Peripheral Neuropathy
Gene therapy shows promise in the management of inherited peripheral neuropathy and the complex array of symptoms that patients experience. Gene therapy holds promise in the management of peripheral neuropathies and in significant improvement of quality of life for patients.
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