New biomarkers of disease activity may help better vet investigational therapies aimed at slowing the insidious neurodegeneration seen in primary progressive multiple sclerosis.
Denali Therapeutics has begun submitting a BLA for tividenofusp alfa as a potential Hunter syndrome treatment, supported by promising biomarker reductions in clinical trials.
An 8 year old boy with genetically confirmed neurofibromatosis type 1 presents with first generalized seizure.