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DTSTAMP:20251216T222015Z
DESCRIPTION:The Annual George Karpati Lecture honours a world-renowned neur
 ologist and clinician-scientist who dedicated his remarkable career to The
  Neuro. Dr. Karpati was celebrated for advancing the diagnosis\, treatment
 \, and understanding of neuromuscular diseases\, especially muscular dystr
 ophy. Each year\, his colleagues host this lecture to highlight recent pro
 gress in neuromuscular research and care\, paying tribute to a beloved fri
 end\, mentor\, and scientific leader.\n\n\nRegister Now\n\n\nThe Era of Be
 spoke Therapies for Genetic Neuromuscular Diseases\n\nLecture Abstract: My
 ology has been an indispensable centerpiece in our expanding understanding
  of basic biology\, cellular physiology\, and genetics over centuries. It 
 has also been a constant partner in development of techniques to study cel
 lular biology\, histopathology\, and disease pathogenesis. However\, many 
 primary disorders of skeletal muscle remain recalcitrant. With the recent 
 advent of DNA- and RNA-directed therapies\, a decades-long vision of treat
 ing patients with rare neuromuscular diseases seems within reach\, althoug
 h many barriers remain to be overcome. This presentation will give an over
 view of monogenic neuromuscular disorders from a physician-scientist viewp
 oint and provide a few concrete examples of approaches in the laboratory t
 o study disease pathomechanisms\, to identify therapeutic targets\, and to
  develop bespoke/precision therapies for these rare diseases.\n\nPayam Moh
 assel\n\n\n\nDr. Mohassel is a physician scientist who specializes in myop
 athies\, muscular dystrophies\, and other hereditary neuromuscular disorde
 rs. He is also the co-director of the Johns Hopkins Myositis Center and a 
 myopathologist. Dr. Mohassel obtained his medical degree from the Johns Ho
 pkins University School of Medicine\, where he remained for a medical inte
 rnship\, residency training in neurology\, and clinical fellowship trainin
 g in neuromuscular medicine. He then joined the National Institute of Neur
 ological Disorders and Stroke Neurogenetics branch as a clinical research 
 fellow under the mentorship of Prof. Carsten Bonnemann for 8 years\, befor
 e returning to Johns Hopkins department of Neurology as faculty. Dr. Mohas
 sel’s research focuses on translational studies in neurogenetic\, neuromus
 cular disorders\, and it spans gene discovery efforts\, mechanistic studie
 s to identify therapeutic targets\, and early phase interventional clinica
 l trials for rare neuromuscular diseases.\n
DTSTART:20260224T210000Z
DTEND:20260224T230000Z
LOCATION:Jeanne Timmins Amphitheatre\, The Neuro
SUMMARY:George Karpati Lecture: The Era of Bespoke Therapies for Genetic Ne
 uromuscular Diseases
URL:/research/channels/event/george-karpati-lecture-er
 a-bespoke-therapies-genetic-neuromuscular-diseases-367516
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