Hagen Cramer

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als ftd als genes als drug precision therapy precision medicine neurodegenerative diseases neurology clinical trials stathmin-2 qrl-201 qrl-101 flexaso platform therapeutic candidates tdp-43 pathology ion channels kv7.2/7.3 pharmacology neuroscience biomarkers anti-sense oligonucleotide motor neuron disease research collaboration disease mechanisms genetic mutations spinal cord neural repair patient stratification epilepsy frontotemporal degeneration gene therapy clinical development cns diseases drug discovery pharmaceutical company translational medicine human neuronal models biomedical research therapeutic targets rna therapeutics nucleic acid therapeutics collaborative research biopharmaceutical healthcare innovation neurobiology peripheral neuropathy clinical safety fda approvals drug candidates molecular biology genomics patients' needs improved treatment outcomes amyotrophic lateral sclerosis frontotemporal dementia antisense oligonucleotides patient-focused innovative therapies neurological disorders disease-modifying treatments kv7 ion channel opener stmn2 expression fragile x syndrome progressive supranuclear palsy medical community engagement research partnerships patient demographics age 40-70 healthcare professionals scientific expertise mission-driven pioneering approach targeted therapies unmet medical needs health care health wellness & fitness hospital & health care biotechnology