If the protein TDP-43 is defective inside a nerve cell it can form neurotoxic aggregates, which are the cause of the ...

Researchers have developed a new therapy that can be injected intravenously right after a heart attack to promote healing and ...

The result is progressive muscle paralysis ... While in healthy cells TDP-43 is mainly found in soluble form in the cell nucleus, in ALS patients it forms poorly soluble aggregates that mainly ...

A potential strategy introduces an entirely new type of therapy capable of addressing previously ‘undruggable’ targets within the cells.

Scientists at Northwestern University and University of California San Diego have developed a new, potent injectable therapy ...

The EC has granted orphan drug designation to Dyne Therapeutics’ DYNE-251 for treating Duchenne muscular dystrophy (DMD).

Autoimmune generalized myasthenia gravis is a disease that manifests with fluctuating muscle weakness. Inebilizumab is a monoclonal antibody that depletes CD19+ B cells, which are central to ...