What is the National Pediatric Myoclonus Center (NPMC)?
The purpose of a national center for myoclonus is to provide the best care possible for patients whose myoclonus began during childhood. This care includes making the correct diagnosis, searching for all reversible (curable) causes, choosing the best drug treatments available, providing information about myoclonus, obtaining psychological and emotional support for the children and their families, and bringing them together with other similar families. We also wish to establish the scope of the problem and increase awareness among federal, private and pharmaceutical agencies. Patients with myoclonus otherwise have no advocate. They are often misdiagnosed, told there is no hope, feel isolated, and are in need of a concerted effort to better their situation.
History of the Center
The National Pediatric Myoclonus Center (NPMC) was founded in 1985 by Dr. Pranzatelli. In 1990 it became a full service center, largely through the efforts of its first research nurse practitioner Elizabeth D. Tate. FNP. C., MN. The membership consists of patients and their families, health care providers, educators and volunteers who support the mission of the organization. Supported by federal, pharmaceutical, institutional, and private funds, it has grown to be a referral center for the U.S., Canada, Mexico, and other countries.
Prof. Dr. Michael R. Pranzatelli, M.D., is a clinician-scientist and Founder and Director of the National Pediatric Myoclonus Center and National Pediatric Neuroinflammation Organization, Inc., whose missions are research, education, and advocacy for children with neuroinflammatory disorders. He is currently Professor of Neurology at University of Central Florida College of Medicine and Adjoint Professor of Neurology at the University of Colorado School of Medicine. A graduate of the Pennsylvania State University, Dr. Pranzatelli completed his pediatric residency at Case Western Reserve University, and his pediatric neurology residency at the University of Colorado. He also completed a three-year basic research fellowship in neuropharmacology at the University of Southern California’s Children’s Hospital. He received the prestigious Young Investigator Award of the Child Neurology Society and is certified both by the American Board of Pediatrics and the American Board of Psychiatry and Neurology with special qualification in Child Neurology. High-ranking institutions where he held faculty appointments include Columbia University in New York City and the George Washington University in Washington, D.C. Dr. Pranzatelli authored 185 publications (150 PubMed) in peer-review medical and scientific journals, presented 89 research meeting abstracts, and given 157 international, national, and local lectures. He has been an ad hoc reviewer for 36 medical and scientific journals. He is consistently listed in Best Doctors in America and has been distinguished by a “Medical Miracles” award and as a “Medical Honoree.” Dr. Pranzatelli has received approximately $4 million in federal, foundation, pharmaceutical, private, and institutional funding for his research, including a Clinical Investigator Development Award from the National Institutes of Health. The Food and Drug Administration Orphan Products Development Program funded some of his clinical drug trials. Dr. Pranzatelli runs the world’s largest center for children with opsoclonus-myoclonus (OMS), an autoimmune neurologic complication of cancer, and is an internationally-recognized authority on the disorder. His continued translational research on pediatric neuroinflammation has identified several biomarkers of disease activity in OMS and lead to biomarker-guided immunotherapy and other innovative treatments.
Elizabeth D. Tate, C-FNP, MN, graduated with distinction from George Mason University School of Nursing. She then earned her Masters of Nursing and became a family nurse practitioner at the University of California at Los Angeles with subsequent national board certification. For the past 20 years, Elizabeth has specialized in pediatric movement/neuroinflammatory disorders, serving as Clinical Director and co-founder of the National Pediatric Myoclonus Center. In 2007 Elizabeth received the national Claire Chee Award for Excellence in Child Neurology Nursing from the Association of Child Neurology Nurses. The award "...recognizes and honors the nurse who has rendered distinguished service within the profession of child neurology nursing, and who demonstrates, through strength of character and competence, a commitment to the care of children and their families with neurological disorders." She has been co-investigator on FDA-sponsored clinical drug trials and multiple research grants for opsoclonus-myoclonus syndrome. She was selected by UCLA Graduate School of Nursing as one of ’60 of the School’s Outstanding Alumni” in 2009, and as one of three healthcare providers by Exceptional Parent or EP Magazine’s ‘2009 Models of Excellence in the Healthcare Profession,” both in 2009. She has lectured for multiple national nursing organizations and published 44 articles in peer-review medical and nursing journals. She is a member of the American Academy of Nurse Practitioners and the International Council of Advance Practice Nurses. Currently, Elizabeth is on the medical board of directors for the non profit National Pediatric Neuroinflammation Organization, Inc., whose missions are research, education, and advocacy for children with neuroinflammatory disorders.
Description of Goals
Our goals are to provide comprehensive, state-of-the-art diagnostic testing and compassionate care, to build a registry of myoclonic disorders of various etiologies to support meaningful research studies, to design and promote clinical and applied basic research in pediatric myoclonus, to provide patient and physician education through the use of newsletters and pamphlets, and to encourage the organization of family support groups. No other center is dedicated to the study of pediatric myoclonus.
Types of Disorders
We evaluate all myoclonic disorders. We specialize in the opsoclonus-myoclonus syndrome and other non-epileptic forms of myoclonus, not myoclonic epilepsy. We do treat many patients with progressive myoclonus epilepsy, since they have both myoclonus and epilepsy. Because myoclonus has so many opsoclonus-myoclonus syndrome possible causes and affects children of any age, race, or ethnic background, we see a diverse patient population.
A Comprehensive Evaluation
To accomplish our goals, we have assembled experts in dozens of disciplines. Some do electrical brain wave studies (EEG and EP), sleep studies (polysomnogram), neuropsychological testing (IQ and other tests of mental function), blood tests for rare metabolic disorders and molecular genetic studies, eye examinations (neuro-ophthalmology), analysis of the chemicals and cells in spinal fluid (neurochemistry and neuroimmunology), measurement of movement (videotaping), determination of the source of myoclonus (back-averaging), and motion analysis studies of brain structure (MRI or CT scan), and advanced studies of brain function (PET or SPECT scan). Insurance companies usually cover the cost of our diagnostic evaluation, and can be contacted in advance for questions.
Sometimes we can arrange a short stay for out-of-state families in our local Ronald McDonald House, which is only a few blocks from the hospital. Call (217) 528-3314.
Patients from out-of-state may take advantage of "miles for kids" flights provided by American Airlines. Pilots donate their time to provide this service. Call (817) 963-8118.
After you leave the clinic, all of the test results will be collected. These results will help your doctor determine the best treatment for your child. If you qualify for a clinical drug trial, the medication will be given to you together with instructions. If you live out-of-town, these materials will be mailed. We will also make arrangements for return visits to us or with your collaborating local physician.
The therapy of myoclonus is as varied as the causes. We try to reverse the underlying brain disorder. If that is not possible, we provide symptomatic treatment. Some reversible causes include infections, immunologic abnormalities, vasculitis, and certain toxic or metabolic disorders.
We draw upon medications, such as new anticonvulsants and antidyskinetic drugs, botulinum toxin injections, immunological therapies, such as IVIG, immunosuppressives, plasmapheresis, and new anti-lymphocyte therapies.
Some patients benefit from speech therapy, physical therapy, counseling, consultation with a dietitian, or dietary supplements.
Finding Solutions through Research
A modern and active research program in pediatric myoclonic disorders is necessary to improve treatment and find eventual cures. Your participation acknowledges your support for our research program and the commitment you share with us of finding better solutions to this difficult problem.
Our center has published many authoritative medical publications on myoclonus. We can provide a list of publications, which are available at medical libraries. We also provide pertinent reprints.
We publish two myoclonus newsletters to communicate new developments about myoclonus. The newsletters discuss common problems and solutions of interest to patients with myoclonus.
Our center offers fact sheets on topics such as drugs to avoid because they worsen myoclonus, recommendations regarding immunizations and profiles on treatment options.
To help educate families and health care professionals about myoclonus, we show videotapes to new patients and their families during your stay. The tapes provide an overview of myoclonus or address a specific myoclonic disorder.
We also started a video consultation network for the purpose of screening videotapes of potential study subjects referred by pediatricians, neurologists, nurses, or self-referred.
Center participants find out about support group activities. People share their feelings about coping with myoclonus and discuss ways to communicate with friends, families, schools, coworkers, and employers about the needs of patients with myoclonus.
Our National Pediatric
Myoclonus Center is listed with the National Organization for Rare Disorders
(NORD), the American Academy of Neurology (AAN), and the National Disease
Research Interchange (NDRI). Through mailings of the Epilepsy Foundation of
America (EFA), the Child Neurology Society (CNS), and journal publications
and lectures, we continue to promote public awareness. For an article on opsoclonus-myoclonus,
see "Friendly Fire" in Discover magazine, April 2000.
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