Format: Medical Meeting

1-7 of 14 Activities
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Live
Medical Meeting
1.5
04/30/2023

Live Stream: PIDD in Pediatric Care: When It’s More Than “Just an Infection”

In this CME Outfitters live-streamed symposium, expert faculty discuss pediatric patient cases that illustrate the timely assessment of telltale signs and symptoms indicative of an underlying immunologic deficiency.

1.5
04/30/2023
6:30 am

Kenneth Paris

Kenneth Paris, MD, MPH, is a Professor of Clinical Pediatrics at Louisiana State University (LSU) Health Sciences Center in New Orleans, where he is also Program Director of the Allergy/Immunology Fellowship Training Program and Division Chief of the Division of Allergy/Immunology. He is also Co-Director of the Jeffrey Modell Diagnostic Center for Primary Immunodeficiency at LSU Health Sciences Center.

Kenneth Paris

Kenneth Paris, MD, MPH, is a Professor of Clinical Pediatrics at Louisiana State University (LSU) Health Sciences Center in New Orleans, where he is also Program Director of the Allergy/Immunology Fellowship Training Program and Division Chief of the Division of Allergy/Immunology. He is also Co-Director of the Jeffrey Modell Diagnostic Center for Primary Immunodeficiency at LSU Health Sciences Center.

Dr. Paris received his medical degree from LSU School of Medicine and his Master of Public Health degree in Applied Health Sciences from the Tulane University School of Public Health and Tropical Medicine in New Orleans. He later completed a residency in Pediatrics at Brown University Warren Alpert Medical School in Providence, Rhode Island, and a fellowship in Allergy and Immunology at LSU Health Sciences Center. Dr. Paris is board certified by the American Board of Allergy and Immunology.

A member of the Clinical Immunology Society and the American Academy of Asthma, Allergy & Immunology, Dr. Paris is the author of book chapters, abstracts, and published articles in such refereed journals as the Annual Review of Immunology, Clinical Immunology, and Nature Immunology.

Kristin Epland

Kristin Epland is a 1998 graduate of the University of Minnesota School of Nursing Family Nurse Practitioner master’s program. She has worked with children and adults with immunodeficiency since 1992 through home infusion nursing, Midwest Immunology Clinic, and currently with Infectious Disease Associates in Minneapolis.

Kristin Epland

Kristin Epland is a 1998 graduate of the University of Minnesota School of Nursing Family Nurse Practitioner master’s program. She has worked with children and adults with immunodeficiency since 1992 through home infusion nursing, Midwest Immunology Clinic, and currently with Infectious Disease Associates in Minneapolis. Her present responsibilities include both inpatient and outpatient care of children and adults with primary immune deficiency and infectious diseases as well as the supervision of nursing and medical services provided through the infusion centers and home infusion. She is a part of the National Nurses Advisory Board of the Immune Deficiency Foundation (IDF).

Kris lives in Maple Grove, Minnesota. In her spare time, she loves traveling, fishing, hockey, and training her two stubborn dogs.

Hey Jin Chong

Dr. Hey Chong is the Division Director of Allergy and Immunology at UPMC Children’s Hospital of Pittsburgh as well as the Medical Director of the Inborn Errors of Immunity Clinic, which she established in 2013. She is also the Director of the Immune Dysregulation Diagnosis and Treatment Center.

Hey Jin Chong

Dr. Hey Chong is the Division Director of Allergy and Immunology at UPMC Children’s Hospital of Pittsburgh as well as the Medical Director of the Inborn Errors of Immunity Clinic, which she established in 2013. She is also the Director of the Immune Dysregulation Diagnosis and Treatment Center.

She received her MD and PhD from the Virginia Commonwealth University Medical College of Virginia, followed by her pediatric residency at the University of Chicago. She completed her fellowship in allergy and immunology at the Brigham and Women’s Hospital and Harvard Medical School.

She then joined the Allergy and Immunology Faculty at UPMC Children’s Hospital of Pittsburgh and University of Pittsburgh School of Medicine, where she is currently an Associate Professor.

 

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Live
Medical Meeting
1.00 Hours
02/23/2023

Live Stream – Manifestations of Gaucher Disease: Rare or Under-recognized?

During this 60-minute symposium live, expert panelists will discuss GD, including its less commonly reported manifestations, diagnostic pathways, and patient-centric multidisciplinary management of the disorder.

1.00 Hours
02/23/2023

Neal J. Weinreb

Neal J. Weinreb, MD, FACP is a graduate of the Jewish Theological Seminary (Bachelor of Hebrew Literature), and SUNY Downstate Health Sciences University (MD, summa cum laude).

Neal J. Weinreb

Neal J. Weinreb, MD, FACP is a graduate of the Jewish Theological Seminary (Bachelor of Hebrew Literature), and SUNY Downstate Health Sciences University (MD, summa cum laude). After a residency in Internal Medicine at Albert Einstein College of Medicine and a fellowship and faculty position at Mt. Sinai School of Medicine, NY, he practiced Hematology and Medical Oncology in South Florida. Dr. Weinreb, the director of the University Research Foundation for Lysosomal Storage Diseases in Boca Raton, FL, retired from clinical practice in May 2018. He now devotes his entire professional attention to clinical research about Gaucher Disease and Fabry Disease. Dr Weinreb is a Voluntary Associate Professor of Human Genetics and Medicine at the University of Miami Miller School of Medicine. Dr. Weinreb is an investigator for the Gaucher and Fabry Registries and a member of the Medical Advisory Board for the National Gaucher Foundation and of the International Working Group for Gaucher Disease.

Dimitri Krainc

Dimitri Krainc, MD, PhD, currently serves as the Ward Professor and Chairman of the Department of Neurology and Director of the Simpson Querrey Center for Neurogenetics at Northwestern University, Feinberg School of Medicine in Chicago.

Dimitri Krainc

Dimitri Krainc, MD, PhD, currently serves as the Ward Professor and Chairman of the Department of Neurology and Director of the Simpson Querrey Center for Neurogenetics at Northwestern University, Feinberg School of Medicine in Chicago. Previously, he spent over two decades at Massachusetts General Hospital and Harvard Medical School where he completed his research and clinical training and served on the neurology faculty. His group discovered how to improve degradation of aggregation-prone proteins such as mutant huntingtin and a-synuclein by the lysosomal pathway. They identified a positive feedback loop between alpha-synuclein and lysosomal glucocerebrosidase in synucleinopathies. Using patient-specific neurons, his lab discovered a link between mitochondrial and lysosomal dysfunction in neurodegeneration of human but not mouse midbrain neurons. Based on these findings, Dr. Krainc’s group initiated the development of activators of lysosomal glucocerebrosidase as targeted therapeutics for Parkinson’s disease. Their recent discovery of direct contacts between mitochondria and lysosomes and organelle dynamics has implications for various neurological and other disorders. He received numerous awards for his work and is an elected member of the Association of American Physicians and the National Academy of Medicine.

Coy Heldermon

Dr. Coy Heldermon is an associate professor of medicine and board-certified medical oncologist at the University of Florida with expertise in the treatment of breast cancer.

Coy Heldermon

Dr. Coy Heldermon is an associate professor of medicine and board-certified medical oncologist at the University of Florida with expertise in the treatment of breast cancer. He is a member of the American Society of Hematology and the American Society of Clinical Oncology. His research expertise is in the use of gene replacement and stem cell therapies for the treatment of inherited disorders such as lysosomal storage diseases.

Tamanna Roshan Lal

Dr. Roshan Lal is a Pediatric Clinical and Metabolic Geneticist working at the Rare Disease Institute, Children's National Hospital in Washington DC.

Tamanna Roshan Lal

Dr. Roshan Lal is a Pediatric Clinical and Metabolic Geneticist working at the Rare Disease Institute, Children’s National Hospital in Washington DC. Her clinical and research interests are geared towards improving the quality of life for children with rare genetic diseases, specifically lysosomal storage diseases and neuro-genetic disorders. Her special interest includes being involved in clinical trials using orphan drugs as well as gene therapy. She is currently the Director of Clinical Trials and Director of International Patient Consultations and Care Referrals for the Rare Disease Institute at the Children’s Hospital.

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On-Demand
Medical Meeting, Webcast
1.5 Hours
06/09/2022

Revisiting IBS: Recrafting the Diagnosis for More Timely Treatment and Patient-Centered Care

This on-demand CME Outfitters symposium features an expert panel discussion crafted with a focus on patients with IBS that provides strategies for obtaining accurate diagnosis, presents new and emerging clinical data on the efficacy and safety of IBS therapies, and details approaches for patient-centered care to achieve optimal outcomes.

1.5 Hours
06/09/2022

Amy Ladewski

Amy Ladewski is a physician assistant within the department of digestive health at Northwestern Memorial Hospital in Chicago, IL.

Amy Ladewski

Amy Ladewski is a physician assistant within the department of digestive health at Northwestern Memorial Hospital in Chicago, IL. She specializes in functional bowel and neurogastromotility disorders, in addition to managing general GI patients. She currently serves as the Vice President of the national non-profit organization GHAPP (Gastroenterology & Hepatology Advanced Practice Providers), with a mission to educate and help advance the careers of GI APPs. Amy serves as an adjunct teacher for the Northwestern Feinberg School of Medicine Physician Assistance program and was recently awarded preceptor of the year. With a background as a registered dietitian, she prioritizes nutrition counseling with her patients. Amy strives to develop collaborative and trusted relationships with her patients to empower them toward better health and wellness.

Lin Chang

Dr. Chang is a Professor of Medicine and Vice-Chief of the Vatche and Tamar Manoukian Division of Digestive Diseases, Department of Medicine at the David Geffen School of Medicine at UCLA.

Lin Chang

Dr. Chang is a Professor of Medicine and Vice-Chief of the Vatche and Tamar Manoukian Division of Digestive Diseases, Department of Medicine at the David Geffen School of Medicine at UCLA. She serves as the Co-Director of the G. Oppenheimer Center for Neurobiology of Stress and Resilience at UCLA. She is also Program Director of the UCLA Gastroenterology Fellowship Program. Dr. Chang’s clinical expertise is in disorders of gut-brain interaction (also known as functional gastrointestinal disorders). Her research focuses on brain-gut interactions underlying irritable bowel syndrome (IBS), specifically, the pathophysiology of IBS related to stress, early life adversity, sex differences, genetic and epigenetic factors, and gut microbiome as well as the treatment of IBS. She has recently served as the Clinical Research Councillor of the AGA Governing Board. Dr. Chang previously served as President of the American Neurogastroenterology and Motility Society (ANMS) and is a member of the Rome Foundation Board of Directors.

William D. Chey

Dr. Chey received a medical degree and training in internal medicine at Emory University School of Medicine. He is currently the Timothy T. Nostrant Collegiate Professor of Gastroenterology at the University of Michigan, Ann Arbor.

William D. Chey

Dr. Chey received a medical degree and training in internal medicine at Emory University School of Medicine. He completed a fellowship in gastroenterology and has remained as faculty at the University of Michigan in Ann Arbor where he is currently the Timothy T. Nostrant Collegiate Professor of Gastroenterology. He holds a joint appointment in the Department of Nutrition Sciences.

Dr. Chey’s research interests focus on the diagnosis and treatment of functional bowel disorders and H. pylori infection. He has mentored more than 50 students, residents, fellows, and junior faculty, many of which are leaders in gastroenterology. He is a medical innovator and holds several patents. Dr. Chey is former Co-Editor-in-Chief of the American Journal of Gastroenterology and has authored more than 350 manuscripts, reviews, chapters, and books. He is a member of the Board of Trustees of the American College of Gastroenterology, Board of Directors of the Rome Foundation, Council of the American Neurogastroenterology and Motility Society, and Board of Directors of the International Foundation of Functional Gastrointestinal Disorders.

Dr. Chey has been elected to “Best Doctors” since 2001 and received the Dean’s Outstanding Clinician Award and Dean’s Award for Innovation and Commercialization at the University of Michigan. He has also received the Distinguished Clinician Award from the American Gastroenterological Association. In 2020 he was awarded honorary membership to the Academy of Nutrition and Dietetics and the prestigious Berk/Fise Award from the American College of Gastroenterology.

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Maintaining Momentum in the Treatment of Chronic Idiopathic Constipation: Framing Practical Clinical Strategies Through Case Studies

In this CME Outfitters OnDemand symposium, expert faculty frame the CIC decision tree with patient cases in order to achieve a confident diagnosis, implement effective treatment plans to improve outcomes, and improve quality of life for patients with CIC.

1.5 Hours
06/02/2022

Lucinda A. Harris

Lucinda A. Harris

Associate Professor of Medicine, Scottsdale, AZ

Lucinda A. Harris, MS, MD, is currently Associate Professor of Medicine, Mayo Clinic Alix School of Medicine and Consultant, Division of Gastroenterology and Hepatology, Mayo Clinic Scottsdale. She graduated from the University of Connecticut School of Medicine. She completed her Internal Medicine Residency at the New York Presbyterian Hospital of Columbia University and her fellowship in Gastroenterology & Hepatology at The New York Hospital /Weill Medical College of Cornell University. She was on the faculty in the Division of Gastroenterology & Hepatology at Weill Cornell Medical School before moving to her current position at Mayo Clinic.

At the Mayo Clinic she has been a past Co-Director of the Motility Group and has led a patient support group there for IBS. Dr. Harris is a current member of the Gastroenterology Fellowship committee and serves as the Department Education coordinator for Gastroenterology, supervising visiting GI fellows and medical students. She has actively been involved in mentoring medical students, residents, and fellows over the years.

Professionally, Dr. Harris is currently President of the Phoenix GI Society. She is also active in professional organizations as a Fellow of the American College of Gastroenterology, the American Gastroenterological Association, and the American College of Internal Medicine. She is also a member of the American Neurogastroenterology and Motility Society. Her special clinical and research interests are in IBS, gastroparesis, chronic constipation, and pelvic floor disorders as well as celiac disease and autonomic disorders. Dr. Harris has written and lectured extensively on these topics.

Brooks D. Cash

Dr. Cash is Chief of the Division of Gastroenterology, Hepatology, and Nutrition and the Dan and Lillie Sterling Professor of Clinical Gastroenterology at the University of Texas McGovern Medical School

Brooks D. Cash

Dr. Cash is Chief of the Division of Gastroenterology, Hepatology, and Nutrition at the University of Texas Health Science Center at Houston, where he is also the Dan and Lillie Sterling Professor of Clinical Gastroenterology at the University of Texas McGovern Medical School and the Endowed Director of the Chao-Ertan Directorship at Memorial Hermann Hospital Texas Medical Center. Dr. Cash received his undergraduate degree in Business Administration (Finance) with Honors from the University of Texas in Austin and earned his medical degree from the Uniformed Services University of Health Sciences in Bethesda, MD. He completed his internship, residency, and gastroenterology fellowship at the National Naval Medical Center in Bethesda, MD, and served for 24 years in the United States Navy, retiring in 2014. Dr. Cash has authored over 200 articles and book chapters on a wide variety of gastrointestinal topics and his clinical interests include disorders of brain-gut interaction, colorectal cancer screening, artificial intelligence, and Barrett esophagus. He was recognized as one of the best gastroenterologists in Houston by Houstonia magazine in 2019 and a Top Doctor by Texas Monthly magazine in 2020 and 2021. In 2020 he was named a Rome Foundation Fellow and was recently appointed to the Rome V Functional Bowel Disorders Committee.

Michael Camilleri

Michael Camilleri

Professor of Medicine, Pharmacology, and Physiology, Mayo Clinic, Rochester, MN

Dr. Camilleri is a consultant in the Division of Gastroenterology and Hepatology at Mayo Clinic in Rochester, Minnesota, and is Professor of Medicine, Pharmacology, and Physiology at Mayo Clinic College of Medicine and Science. His research interests include clinical enteric neurosciences, gut neurohormonal control, obesity, irritable bowel syndrome, and pharmacology and pharmacogenomics; his work is funded by National Institutes of Health. He has received numerous awards and honors including the 2012 Ismar Boas Medal from the German Society of Digestive and Metabolic Disease, the 2012 American Gastroenterological Association (AGA) Distinguished Mentor Award, the Janssen Research Award, the AGA Joseph B. Kirsner Award, the AGA Julius Friedenwald Medal in 2021, as well as honorary doctorates from the University of Antwerp (Belgium) and the University of Malta, and he was elected to the membership of the prestigious Association of American Physicians. Dr. Camilleri participates in educational and mentorship activities at Mayo Clinic and has mentored more than 80 national and international postdoctoral fellows and scientists. He maintains an active clinical practice in gastroenterology at Mayo Clinic, specializing in gastrointestinal motility disorders, gastroparesis, and functional GI disorders. He is Past-President of the American Neurogastroenterology and Motility Society and the American Gastroenterological Association as well as former editor of Clinical Gastroenterology and Hepatology and Neurogastroenterology and Motility.

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On-Demand
Medical Meeting
1.50 Hours
05/04/2022

Beneath the Surface: Moving Beyond Symptomatic Control in Obstructive Hypertrophic Cardiomyopathy

In this CME Outfitters OnDemand symposium from Heart Rhythm 2022, three experts in HCM explore the latest strategies and updates in HCM diagnosis and management, including progress with disease-specific treatments that target cardiac myosin.

1.50 Hours
05/04/2022
Carolyn Yung Ho, MD

Carolyn Yung Ho

Carolyn Ho is an Associate Professor of Medicine at Harvard Medical School and Medical Director of the Cardiovascular Genetics Center at Brigham and Women's Hospital. She obtained her undergraduate degree from Yale University and her medical degree from Harvard Medical School. Dr. Ho trained at Brigham and Women's Hospital for both Internal Medicine residency and Cardiology fellowship before joining the staff of the Cardiovascular Division in 2001.
Carolyn Yung Ho, MD

Carolyn Yung Ho

Carolyn Ho is an Associate Professor of Medicine at Harvard Medical School and Medical Director of the Cardiovascular Genetics Center at Brigham and Women’s Hospital. She obtained her undergraduate degree from Yale University and her medical degree from Harvard Medical School. Dr. Ho trained at Brigham and Women’s Hospital for both Internal Medicine residency and Cardiology fellowship before joining the staff of the Cardiovascular Division in 2001.

Dr. Ho’s research focuses on characterizing early phenotypes of sarcomere mutations in inherited cardiomyopathies, leading a large, multicenter registry of genetic cardiomyopathies, and developing clinical trials to diminish the progression of hypertrophic cardiomyopathy. The overall goal is to use genetic/mechanistic insights, careful clinical study, and collaboration to improve the care of patients and families with genetic heart disease.

Steve R. Ommen, MD, FACC, FAHA

Steve R. Ommen

Dr. Steve R. Ommen is a Professor of Medicine at Mayo Clinic College of Medicine and a Consultant in the Department of Cardiovascular Medicine. He serves as the Director of the Mayo Hypertrophic Cardiomyopathy Clinic, the largest and longest-standing dedicated HCM Center in the world
Steve R. Ommen, MD, FACC, FAHA

Steve R. Ommen

Dr. Steve R. Ommen is a Professor of Medicine at Mayo Clinic College of Medicine and a Consultant in the Department of Cardiovascular Medicine. He serves as the Director of the Mayo Hypertrophic Cardiomyopathy Clinic, the largest and longest-standing dedicated HCM Center in the world. Mayo’s HCM clinic is recognized as a leader in clinical outcomes, research, and patient education. Dr. Ommen is also Co-director of the Structural Heart Disease program, Medical Director for AskMayoExpert, and Medical Director of the Center for Connected Care.

Dr. Ommen specializes in caring for patients with structural heart disease with a special emphasis on valvular heart disease and hypertrophic cardiomyopathy (HCM). He directs Mayo Clinic’s Hypertrophic Cardiomyopathy program, which is the world’s largest and longest standing program in this disease. He is a requested speaker globally and helped write international guidelines on the appropriate management of patients with HCM.

Dr. Ommen leads Mayo Clinic’s telemedicine team, a critical component in Mayo Clinic’s strategy to provide comprehensive care with a seamless patient experience. He oversees the development of products, services, and systems that bring health care to patients where they need it.

Additionally, he is active in advocating for the advancement of connected care through discussions and presentations at regional, state, and national levels so that these advancements in health care delivery can be made available to people and patients more broadly.

Dr. Ommen has earned many awards throughout his career including the Outstanding Achievement Award in Clinical Cardiology, the Mayo Award of Individual Excellence, the Mayo Award for Team Excellence, the Excellence in Teaching Recognition for Mayo Clinic College of Medicine, and the Outstanding Course Director Award from the Mayo School of Continuing Medical Education. He has been recognized as one of the top 15 Digital Health care Executives.

Dr. Ommen received his Bachelor of Science degree in Biomedical Engineering from Northwestern University. His undergraduate medical education, Internal Medicine Residency and Cardiovascular Diseases Fellowship were all completed at Mayo Clinic, Rochester, Minnesota.

Theodore Abraham, MD, FACC

Theodore Abraham

Dr. Abraham is the Meyer Friedman Distinguished Professor of Medicine and Co-Director of the Hypertrophic Cardiomyopathy Center of Excellence at University of California at San Francisco. He is a board- certified cardiologist who is recognized for his expertise and contributions in Hypertrophic Cardiomyopathy and Advanced Echocardiography.
Theodore Abraham, MD, FACC

Theodore Abraham

Dr. Abraham is the Meyer Friedman Distinguished Professor of Medicine and Co-Director of the Hypertrophic Cardiomyopathy Center of Excellence at University of California at San Francisco. He is a board- certified cardiologist who is recognized for his expertise and contributions in Hypertrophic Cardiomyopathy and Advanced Echocardiography. He has had an HCM- centered clinical practice for the last 19 years across 3 institutions starting at the Mayo Clinic Rochester, Johns Hopkins and now at UCSF. Previously he served as the Founder- Director of the Johns Hopkins HCM Center of Excellence. His particular clinical interest is in the management of complex obstructive HCM and the use of advanced imaging in the diagnosis and monitoring of HCM patients. His clinical research program has been a leading site for clinical trials for emerging drug therapies and he serves on the steering and/or publications committees for some of these trials. He has over 150 peer reviewed publications focused on HCM, computational flow dynamics and deep learning, and has had continuous NIH and other research funding for over 20 years. He received the 2019 Richard Popp Teaching and Mentorship Award from the American Society of Echocardiography. He served or serves on the editorial boards of the JACC, JACC Cardiovascular Imaging, JASE and Circulation Imaging.

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On-Demand
Medical Meeting
0.00 Expired
04/16/2021

Evidence-Based Strategies to Facilitate Access to Therapies for Patients with Spinal Muscular Atrophy

This enduring material focuses on implementing evidence-based strategies for diagnosing SMA, applying the latest clinical evidence of approved and emerging therapies.

0.00 Expired
04/16/2021
8:00 am

Erin Neil

Dr. Erin Neil is a Clinical Assistant Professor of Pediatrics at the University of Michigan, where she is the Director of the Pediatric Neuromuscular Program and Co-Director of the Muscular Dystrophy Association Clinic.

Erin Neil

Dr. Erin Neil is a Clinical Assistant Professor of Pediatrics at the University of Michigan, where she is the Director of the Pediatric Neuromuscular Program and Co-Director of the Muscular Dystrophy Association Clinic. She serves as the Medical Director for the State of Michigan’s SMA Newborn Screening program, which includes administrative and clinical oversight for care of newborns diagnosed with SMA.

Dr. Neil completed medical training at the Kansas City University, College of Osteopathic Medicine; pediatric neurology training at the Detroit Medical Center/Wayne State University/Children’s Hospital of Michigan; and Neuromuscular fellowship at University of Texas Southwestern, Dallas.

Crystal M. Proud

Dr. Proud is the director of the Neuromuscular Clinic and Spinal Muscular Atrophy Center at Children's Hospital of The King's Daughters (CHKD).

Crystal M. Proud

. Proud is the director of the Neuromuscular Clinic and Spinal Muscular Atrophy Center at Children’s Hospital of The King’s Daughters (CHKD), working collaboratively to provide a comprehensive approach to patients with neuromuscular diseases. She attended college at the University of Virginia where she majored in Neuroscience, and then studied at Eastern Virginia Medical School where she received her Doctorate of Medicine. Dr. Proud pursued general Pediatrics training in Atlanta, Georgia at Emory University and then trained in Neurology to receive special qualifications in Child Neurology at Stanford University. She remained at Stanford for her Neuromuscular Medicine fellowship and then returned home to Virginia to join her current practice as part of Children?s Specialty Group, serving the patients at CHKD in Norfolk, Virginia. During her subspecialty training at Stanford University, Dr. Proud participated in landmark clinical trials including one that supported FDA approval of the first treatment for spinal muscular atrophy (SMA). She has continued this interest at CHKD where she now serves as primary investigator for several clinical trials evaluating novel treatments for patients with various neuromuscular conditions. Her research focus is in the development and conduct of therapeutic trials for SMA and Duchenne muscular dystrophy. Dr. Proud serves as the medical director for the CHKD Novel Therapeutics and Gene Therapy Center, with a commitment to optimizing clinical care and offering participation in clinical research to patients and families with rare disease.