John Najim

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biotechnology: pharmaceutical preparations health care biotechnology research biotechnology nucleic acid therapies rare diseases muscle disease force platform muscle-targeted therapies myotonic dystrophy duchenne muscular dystrophy facioscapulohumeral muscular dystrophy clinical trials antibody engineering oligonucleotide therapeutics antisense oligonucleotide exon skipping gene therapy genetic diseases orphan drug designation functional dystrophin patient advocacy muscle function restoration fda european medicines agency drug development tissue delivery safety & efficacy pediatric diseases disease-modifying therapies community engagement patient access patient stories genetic mutation oligonucleotide design therapeutic payload receptor biology biopharmaceuticals metabolic muscle diseases research collaborations medical innovations therapy design clinical program muscle biology caregiver support community insights patient engagement pharmacodynamics biomarkers clinical endpoints muscle-related genetic disorders healthcare resource utilization clinical-stage therapeutics genetically driven diseases neuromuscular diseases precision medicine investigational therapeutics fda fast track designation patient outcomes innovative treatments healthcare professionals patient communities partnerships collaborations muscle health therapeutic delivery oligonucleotide therapies unmet medical needs functional improvement regulatory approval transformative therapies genetic causes serious muscle diseases health wellness & fitness hospital & health care