Nature3mon
ARSACS, a spastic ataxia common in northeastern Québec, is caused by mutations in a new gene encoding an 11.5-kb ORF
The resulting products were purified and sequenced as above. Bouchard, J.-P. Recessive spastic ataxia of Charlevoix-Saguenay. in Handbook of Clinical Neurology 16: Hereditary Neuropathies and ...
University of Sheffield4d
Sheffield spinout takes major step towards advancing pioneering treatments for Hereditary Spastic Paraplegia (HSP)
Today, the company announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for its novel adeno-associated virus (AAV) gene therapy ...