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All agents approved for marketing must meet the regulatory standards of the country in which they will be marketed, and drug development programs are designed to meet these regulatory requirements. There are frequent interactions between regulators and sponsors during the development and trial process. The FDA in the USA, the EMA in Europe, the NMPA in China, and the Japanese PMDA and other national regulatory bodies communicate regularly and share information. Regulators promote drug development for serious diseases with unmet needs such as Alzheimer’s disease (AD) and can use regulatory tools to facilitate the drug development process. Regulatory agencies oversee the manufacture of treatments (drugs, monoclonal antibodies) in addition to supervising the development process through clinical trials. Regulatory science and regulatory specifications evolve in concert with new knowledge. Agencies now recognize mild cognitive impairment and preclinical AD as populations where therapeutic indications could apply. Agencies have integrated biomarkers into their concepts of drug development and have specified approaches to biomarker qualification.
Progressive myoclonus epilepsy of the Unverricht-Lundborg type (EPM1) is an autosomal recessive neurodegenerative disorder that has the highest incidence among the progressive myoclonus epilepsies worldwide. The primary therapeutic approaches for EPM1 patients include rehabilitation and symptomatic pharmacologic management. Pharmacologic intervention includes: valproic acid, clonazepam, high doses of piracetam, levetiracetam and topiramate and zonisamide. Several case reports suggest that treatment of EPM1 patients with the antioxidant N-acetylcysteine (NAC) alleviates key features of the disorder including dysarthria, ataxia, and seizures. Although the role of oxidative stress in EPM1-linked neuronal degeneration is not completely understood, a specific decrease in cerebellar defenses against oxidative stress and a concomitant increase in lipid peroxidation occurs in the mouse model for EPM1. Myoclonic seizures can also be easily misdiagnosed as tonic-clonic seizures or even pseudoepileptic seizures, especially as the majority of the myoclonic movements are not time-locked to electroencephalogram (EEG) discharges.