Hereditary spastic paraplegia (HSP) is the name given to a group of inherited neurological disorders characterized by progressive stiffness or tightness of the muscles (spasticity) in the lower limbs.
Elena Rugarli and colleagues from the National Neurological Institute in Milan have used gene therapy to save sensory and skeletal muscle nerve fibers from degeneration in mice with hereditary spastic ...
The US Food and Drug Administration has cleared BlackfinBio's investigational new drug (IND) application for a Phase I/II trial of BFB-101, an adeno-associated virus gene therapy designed to treat ...
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