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Muscular Dystrophy

mdstarnet logo The Muscular Dsytrophy Surveillance Tracking and Research Network (MD STARnet) is a research network funded by the Centers for Disease Control and Prevention. There are six sites in the network. They are Arizona, Colorado Hawaii, Georgia, Iowa, and western New York. The program started in the year 2003. It will continue until at least 2010.

Duchenne/Becker muscular dystrophy (DBMD) is the most common muscular dystrophy in children. DBMD affects about 1 out of every 3,500 to 5,000 boys. Girls rarely have DBMD. Of the more than 4 million births in the United States each year, about 400 to 600 are children with DBMD.

MD STARnet’s goal is to find everyone with Duchenne or Becker muscular dystrophy born after 1981 in these sites. This will help us estimate the number of people who have DBMD. Another goal is to gather information about people who have DBMD. This includes how DBMD was diagnosed, signs and symptoms of DBMD, and the services and treatment patients and families receive.

We hope to answer questions such as:

How common is DBMD?
Is it equally common in different racial and ethnic groups?
What are the early signs and symptoms of DBMD?
Do factors such as the type of care received or the type of gene changes affect the severity or course of DBMD?
What health care-related services, medical and social, are families receiving?
Does health care vary in different areas? Do different populations receive different care?

Children with DBMD will be identified using information gathered from many different sources such as clinical medical records and hospital records. MENTION ANY OTHERS?? Public health researchers will collect information from these sources to keep track of each child’s health care and changes over time. All of the data collected will be pooled anonymously (without names).

Families will also be invited to take part in interviews and surveys. The purpose of these interviews and surveys is to identify and describe the health and service needs of individuals and families affected by DBMD that might not be found in the medical records. We hope that knowledge gained from this study will lead to improved health care and support systems in the future. This information will be shared with doctors, public health officials, therapists, and educators. This could help families receive better health care. It could also help the organizations that serve families better meet their needs.

Publications and Findings

MD STARnet research has been presented at many scientific meetings. Presentations have been made at the American Academy of Neurology and the World Muscle Society. We are doing research on gene problems that cause DBMD, the prevalence of DBMD across our sites, steroid use, and the diagnostic journey for families.

In 2006, MD STARnet published: The Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet): Surveillance Methodology. Birth Defects Research (Part A), 2006, issue 76, pages 793-797. Lisa A. Miller, Paul A. Romitti, Christopher Cunniff, Charlotte Druschel, Katherine D. Mathews, F. John Meaney, Dennis Matthews, et. al.

In this report, we described our method for finding persons with DBMD and gathering information about them and their medical care. We develop a standard tool for recording and tracking information over time. Our sources for information go beyond the usual sources such as neuromuscular clinics and neurologists’ offices. We included service sites for children with special health care needs, hospitals, and hospital discharge databases. We also developed a way to confirm each diagnosis of DBMD. Neuromuscular doctors from each state meet and decide if a case meets the definition of DBMD. This system is critical to our goal of understanding how many people have DBMD and their health outcomes.

MDStar.net Sites

map of sites Arizona
Arizona MD STARnet is led by two groups: 1) the Section of Medical and Molecular Genetics in Pediatrics at University of Arizona College of Medicine; and 2) the University Center on Disabilities at the University of Arizona. Chris Cunniff, MD, is the Principal Investigator. Jennifer Andrews, MBA, is the Program Coordinator. The team works on many DBMD projects. One study looks at the kinds of DNA testing used to diagnose DBMD. Another study looks at heart function in children with DBMD. The researchers are almost finished with a project on the use of devices that improve independence and quality of life. This is called assistive technology.

Colorado
The Colorado Department of Public Health and Environment and The Children’s Hospital in Denver work together on MD STARnet. Lisa Miller, MD, MSPH, and Dennis Matthews, MD, are the Principal Investigators. Dr. Miller leads the Surveillance and Research Committee of MD STARnet. Dr. Matthews heads the Clinical Review Committee. MD STARnet is part of the Colorado Responds to Children with Special Needs program in the public health department. This program studies and helps to prevent conditions that affect children. These conditions include a variety of birth defects, autism, fetal alcohol syndrome, and muscular dystrophy. The Children’s Hospital Muscle Clinic in Aurora serves children and teens. The clinic is studying the use of Alendronate to treat weak bones in boys with Duchenne muscular dystrophy (DMD). Bone fractures are common in boys with DMD as they get older and as a result of steroid use. The goal is to determine if Alendronate with vitamin D and calcium improves bone mineral density and reduces the number of bone fractures in boys with DMD.

Hawaii

This is a collaborative project involving representatives from Department of Health Programs (Birth Defects, Children with Special Health Needs, and Early Intervention Services), Muscular Dystrophy Association (MDA) clinics, Shriners Hospital for Children, Tripler Army Medical Center, Kapiolani Medical Center, Queen’s Medical Center, private neuromuscular clinics, and other pediatric and neurological practices located in Hawai‘i and Guam. Sylvia Au, M.S., C.G.C., is the Director of the Hawaii MD STARnet project. The aim of this project is to increase the number of Asian and Pacific Island participants within the existing study population. Data gathered by project staff will characterize the complications and long term outcomes of individuals with DBMD who are geographically isolated and may face unique barriers. In addition, the project will describe the history and outcome of individuals with DBMD who receive different levels of medical and preventative care while developing a long term monitoring system for these families. Finally, the culture of Hawai`i and Guam may provide insight into unique methods of caring for children and families with DBMD.

Iowa
The Iowa MD STARnet program is based at the University of Iowa. Staff members come from the Colleges of Public Health, Medicine, and Nursing. Paul Romitti, PhD, and Katherine Mathews, MD, lead the program. Dr. Romitti has experience in monitoring and database management. Dr. Mathews is an expert in neuromuscular diseases in children. Iowa serves as the data coordinating center for MD STARnet. They are interested in quality of life, psychosocial adjustment, and impact of DBMD on the family. Iowa conducted a pilot Needs Assessment in their state. They are taking the lead in finalizing this tool for use throughout the MD STARnet population.

New York
The New York State Department of Health and the University of Rochester Department of Neurology work together on New York MD STARnet. Charlotte Druschel, MD, MPH, and Shree Pandya, PT, MS, are the Principal Investigators. Dr. Emma Ciafaloni is the MD STARnet clinician. Dr. Richard Moxley provides guidance for the New York MD STARnet program. Deborah Fox, MPH, is a Co-Investigator from the New York State Department of Health. The New York team is in charge of training staff to collect information from medical records. They lead quality monitoring for the network. They also study delays in diagnosis for boys with DBMD. They hope to share their findings with doctors, physical therapists and specialists to help reduce such delays. The group is interested in care patterns across states for patients with DBMD. They continue to look at benefits and side effects of long term steroid treatment.

Georgia
CDC is working with the Georgia Division of Public Health. Together they run the Georgia MD STARnet program. Georgia was added as the fifth MD STARnet state in 2006. One reason Georgia was chosen as a site was to add diversity to the MD STARnet population. They are interested to see if there are differences in treatment and services among ethnic and racial groups. It is important to find all the people who have DBMD in Georgia to find out how common DBMD is. So far the Georgia team has found many people with DBMD by working with larger clinics in Georgia, including those in Atlanta, North Georgia, and Augusta. Their next goal is to find people with DBMD in South Georgia. These people might get care from smaller clinics in Georgia or from larger clinics in nearby states like Florida. If you live in Georgia and want to be a part of MD STARnet, please contact the Georgia MD STARnet study coordinator.

Resources

Family and Patient Organizations

Muscular Dystrophy Association (MDA) is a health agency made up of scientists and volunteers. MDA offers services like medical exams, flu shots, support groups, and summer camps for kids. They also help with purchase of wheelchairs and leg braces. For more information, visit their website.

Muscular Dystrophy Association In Colorado
720 South Colorado Boulevard, 480-S
Denver, CO 80246
303-691-3331
Fax: 303-691-3379
Email: denvereast@mdausa.org

To register with MDA if you have a neuromuscular disease, make a clinic appointment and find out more information about other services for people and families affected by a neuromuscular disease in MDA's program, please contact MDA in Colorado.

Muscular Dystrophy Family Foundation (MDFF) was founded in the year 1958. They provide adaptive equipment. This includes wheelchairs, hospital beds, van lifts, ramps, shower chairs, communication devices, and breathing machines. They also offer support services, such as clinical programs, home visits, and social outings. For more information, go to their website.

Parent Project Muscular Dystrophy (PPMD) was founded in 1994 by parents of children with DBMD. Their mission is to improve treatment, quality of life, and long term outlook. Their website offers information about treatment and personal stories. They have a conference every summer where doctors, scientists, and parents learn about research, care, and advocacy issues.

Finding Clinical Trials is a government website, which provides a list of current federal and private clinical trials. You can search for trials related to DBMD. You will find information about studies, who may join, locations in the United States, and phone numbers for more details.

Kid-Friendly Web Sites

Kids Health is a website that gives doctor-approved health information about infants, children, and teens. The web site offers resources for kids, teens, and parents. For more information, visit their website.

Kid Quest on Disability and Health is a CDC-sponsored web site for middle school students. It gets kids thinking about people with disabilities. Parents and teachers can change the materials to meet students' learning styles and levels. The topics can be used in lessons about health, social studies, and tolerance in society. Parents and teachers can work with students to share ideas, talk about issues, and encourage reflection. Students can use the site on their own, too. For more information, visit their website.

Health Care

The Children’s Hospital Rehabilitation Clinic
13123 East 16th Avenue;
Aurora, CO 80045
720-777-2806

The primary goal of The Children’s Hospital Rehabilitation program is to optimize each child’s abilities to adapt to the environment, to ease the effects of disability on the child’s development, and to allow the child to be fully included in community living. For more information, call the number above or visit their website.

Insurance Coverage

Patient Advocate Foundation (PAF) is a national nonprofit organization. Their goal is to protect patient rights. They help solve insurance, job, and debt matters related to health conditions. Visit their website for more information.

Genetics Information

Dolan DNA Learning Center helps people understand genetics:

DNA from the Beginning - Lessons on DNA, genes and heredity.
Your Genes and Your Health - This website has a section on Duchenne and Becker muscular dystrophy.

Understanding Gene Testing is a website that gives basic information about gene testing. It answers common questions about the science, possible benefits, and possible risks of gene testing. Visit their website for more information.

If you have any questions about MD STARnet in Colorado please contact:
April Montgomery, MHA - Project Coordinator
Colorado Responds to Children with Special Needs
Colorado Department of Public Health and Environment
4300 Cherry Creek Drive South
Denver, CO 80246
303-692-2620

Project Staff

Principal Investigator:

Lisa Miller, MD, MSPH
Director, Disease Control and Environmental Epidemiology Division
Colorado Department of Public Health and Environment

Co-Principal Investigator:

Dennis Matthews, MD
Chairman and Professor
Department of Rehabilitation Medicine
University of Colorado Denver Health Sciences Center
The Children’s Hospital

Local Clinical Reviewer:

Joyce Oleszek, MD
The Children’s Hospital

Project Coordinator:

April Montgomery, MHA

Data Manager:

Jennifer Donnelly, BS

Monitoring Specialists:

Cynthia Vogel, RHIA, MS

Beverly Meek, RHIA