The Effect of Dalfampridine in Hereditary Spastic Paraparezi: Randomized Controlled Trial
Status:
Not yet recruiting
Trial end date:
2023-01-30
Target enrollment:
Participant gender:
Summary
Hereditary spastic paraplegias (HSPs) are characteterised by progressive lower extremity
spasticity and weakness. The signs and symptoms of HSP are consequences of retrograde
degeneration of the longest nerve fibres in the corticospinal tracts and posterior columns.
There is a clinically and genetically heterogenity with more than 70 identified causative
genes. HSP can be inherited as autosomal dominant , autosomal recessive, X-linked or maternal
(mitochondrial) trait. To date, there are no effective treatments that can slow or halt the
progressive disability or reverse the neurodegeneration in HFS. Treatment is symptomatic and
includes physical therapy, oral antispastic drugs, botulinum toxin therapy, and intratechal
baclofen pump implantation.