Medicine Grand Rounds: The RCT is Dead; Long Live the RCT @ LKSC, Berg Hall
Feb 28 @ 8:00 am – 9:00 am
Medicine Grand Rounds: The RCT is Dead; Long Live the RCT @ LKSC, Berg Hall | Palo Alto | California | United States

Presenter: Derek Angus, MD, MPH, FRCP
Chair and Professor, Department of Critical Care Medicine
University of Pittsburgh

Derek Angus, MD, is Professor and Chair of the Department of Critical Care Medicine and Director of the Clinical Research, Investigation, and Systems Modeling of Acute Illnesses (CRISMA) Center at the University of Pittsburgh. He holds secondary appointments in Medicine, Health Policy and Management, and Clinical and Translational Science. A graduate of the University of Glasgow and University of Pittsburgh, he also spent a year in Hong Kong with Médecins Sans Frontières.

Angus’ research interests include clinical, epidemiologic and translational studies of sepsis, pneumonia, and multisystem organ failure and health services research of the organization and delivery of critical care services. He has led several large NIH-funded multicenter studies in the critically ill, the most recent of which is ProCESS (Protocolized Care for Early Septic Shock), a 40-center study focusing on how to best provide early resuscitation for septic shock. Angus has published several hundred papers, reviews, and book chapters, is currently section editor for “Caring for the Critically Ill” for JAMA, and is the recipient of numerous awards, including the 2009 American College of Critical Care Medicine Distinguished Investigator Award.

Medicine Grand Rounds: Antisense controls neurodegeneration @ LKSC, Berg Hall
Mar 7 @ 8:00 am – 9:00 am
Medicine Grand Rounds: Antisense controls neurodegeneration @ LKSC, Berg Hall | Palo Alto | California | United States

Presenter: John Day, MD, PhD
Professor of Neurology, Genetic Pediatrics, and, by courtesy, Pathology
Stanford University

John Day, MD, PhD, works with the Neuromuscular Division, which organizes a comprehensive effort to combat and conquer diseases of the peripheral nerves and muscles, including the muscular dystrophies (myotonic, Duchenne, limb girdle, facioscapulohumeral, and congenital muscular dystrophies), motor neuron disorders (ALS and SMA), neuromuscular junction disease (MG, CMS), and peripheral neuropathies (CMT, CIDP). While keeping the patients and families foremost in mind, their research seeks to: define and understand genetic causes; clarify the molecular and cellular consequences of genetic change; determine the multisystemic features that are underappreciated but clinically significant consequence of these diseases; develop and improve methods for managing and treating each disease.

They have identified the genetic cause of several neuromuscular disorders, most notably myotonic dystrophy type 2, which they continue to study to advance understanding of all forms of myotonic dystrophy. They have also contributed to genetic understanding of Duchenne muscular dystrophy, and other muscle and ataxic disorders. They are continuing to investigate the epigenetic and molecular consequences of these diseases through investigation of patient-derived specimens.

They have focused on defining the central nervous system features of neuromuscular disorders, which severely impact patients and families but have been incompletely investigated, explained or managed. Detailed neuropsychological and brain MRI studies are helping to define the developmental and progressive CNS aspects of these conditions, for which they then seek molecular and cellular explanations through cell-based studies of patient-derived specimens.

To assure their research is translatable to clinical practice, they are simultaneously involved in collaborative clinical research on novel treatments for neuromuscular disease, including antisense oligonucleotides and pharmacologic manipulation of muscle function, viral gene therapies and cell-based treatments.

In summary, they work with patients to define neuromuscular disorders more rigorously and understand them more thoroughly, so novel treatments will successfully combat these devastating disorders.