Diseases, Conditions, Syndromes

'Very promising' treatment for Huntington disease discovered

Medical researchers at the University of Alberta have discovered a promising new therapy for Huntington disease that restores lost motor skills and may delay or stop the progression of the disease based on lab model tests, ...

Neuroscience

ALS neuron damage reversed with new compound

Northwestern University scientists have identified the first compound that eliminates the ongoing degeneration of upper motor neurons that become diseased and are a key contributor to ALS (amyotrophic lateral sclerosis), ...

Medical research

Calcium: A key player for a promising and safe brain treatment?

A promising therapy for a range of brain diseases involves antisense oligonucleotides (ASOs)—specialized molecules that can modulate RNA and alter protein production—directly injected into the cerebrospinal fluid, in ...

Genetics

New insights into the origins of spinal muscular atrophy

Columbia researchers have discovered how a genetic defect leads to spinal muscular atrophy (SMA), a critical piece of information about the disease that neurologists have been seeking for decades.

Medications

Potential drug targets for ALS and FTD identified in two studies

A pair of collaborative studies led by Fen-Biao Gao, Ph.D., have identified two potential drug targets for the diseases amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The studies, which appear in Nature ...

Neuroscience

Stem cell-gene therapy shows promise in ALS safety trial

Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. This combined stem cell and gene therapy can potentially ...

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