Research
The following is a list of the research activities of the Neuromuscular Disease Center:
On-going clinical studies:
***New-Starting soon***Etanercept in Dermatomyositis
Etanercept is one of a class of drugs which alter levels of a naturally occurring chemical in the body called Tumor Necrosis factor alpha (TNF-a). TNF-a is an important part of the body's inflammatory response. Etanercept is currently approved by the FDA for use in patients with inflammatory diseases such as Rheumatoid arthritis. It is believed that this drug will also benefit people with dermatomyositis, a neuromuscular disorder that has similarities to an inflammatory disease.
The primary objective of the study is to assess the safety and tolerability of etanercept in patients with Dermatomyositis .
For more information, please contact Eric Graf, at the Neuromuscular Disease Research Center at 503-418-2124 or neuromus@ohsu.edu for more information.
Pompe Disease
***New*** Multinational, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Myozyme, Recombinant Human Acid alpha-Glucosidase (rhGAA), Treatment in Patients with Late-Onset Pompe Disease (IRB#e2475)
Pompe Disease is a condition in which the body is unable to produce an enzyme (Acid alpha-Glucosidase) which is essential in the normal function of muscles. Patients suffer from muscle weakness and in the later stages breathing difficulties.
Myozyme is an enzyme replacement therapy which seeks to increase the levels of Acid alpha-Glucosidase in the body. This drug was recently approved by the FDA for the treatment of the infantile version of Pompe Disease.
For more information, please contact Eric Graf, at the Neuromuscular Disease Research Center at 503-418-2124 or neuromus@ohsu.edu for more information.
Lambert-Eaton Myasthenic Syndrome
***Now open***Compassionate use of 3,4-diaminopyridine in Lambert Eaton Myasthenic Syndrome (IRB#e1965)
A drug known as 3,4-diaminopyridine (DAP for short) has been found safe and effective for people with Lambert-Eaton myasthenic syndrome (LEMS), a form of myasthenia (fluctuating weakness) that involves loss of the chemical signal that normally flows from nerve to muscle cells.
Numerous research studies have shown that subjects who received DAP had a significantly greater improvement in their muscle function and in the electrical activity recorded from their muscles. DAP has few side effects, but has not been approved by the FDA and is not available for commercial use. OHSU has applied to the FDA for permission to use the drug on a compassionate basis. Any patient with LEMS is eligible to receive DAP.
For more information, please contact Eric Graf, at the Neuromuscular Disease Research Center at 503-418-2124 or neuromus@ohsu.edu for more information.
Muscular Dystrophy
***NEW (Now open)*** Heart Health Matters in DBMD Carriers Too (eIRB# 2096)
Research data suggests that up to 10% of DBMD carriers are at an increased risk for serious heart problem. The purpose of this survey is to collect information about the heart health knowledge,
beliefs, and behaviors of women who either carry a genetic change associated with
Duchenne or Becker Muscular Dystrophy (DBMD) or have a son with DBMD, or both. Heart
health is important for everyone. Heart disease is the number one cause of death of men
and women in the United States, but there are ways to promote heart health. This survey
focuses on the heart health of women who carry a genetic change associated with DBMD
because these women can have heart problems that may be related to being a DBMD
carrier. The results will be used to design heart health education messages for these
women. This survey asks questions about you, your health and your family history, what
you know about heart disease, and your feelings about the effect of DBMD on you and your
family.
The study is being done by the Centers for Disease Control and Battelle Centers for Public health research and Evaluation.
If you are a women who either carries the genetic change associated with Duchenne or Becker Muscular Dystrophy (DBMD) or have a son with DBMD, and are interested in participating in this survey, please contact Eric Graf, at the Neuromuscular Disease Research Center at 503-418-2124 or neuromus@ohsu.edu for more information.
***New (Beginning soon)*** National Initiative for Families with Duchenne (eIRB# 2097)
This is the first large scale study of the needs of the DMD community in the United States. In this study, we will look at the difficulties that families face in getting the services that they need. The goal of the study is to help make living with DMD easier. To do this, we need to hear directly from you. This nationwide survey is sponsored through a cooperative agreement of the Centers for Disease Control and Prevention and the Association of American Medical Colleges. It is being carried out by the Cooperative International Neuromuscular Research Group and Columbia University. This project is being done with the cooperation of the Duchenne muscular dystrophy community.
The survey was designed specifically for families with DMD. It asks questions about your family life and how your family is coping with the illness. It also asks what type of clinic is in your area, which types of doctors your family sees, and what other services are offered in your area. We will compare all of the answers we get from families across the country. We know that you are an expert in living with DMD since you do it every day. We hope that you will share your experiences to help us learn more about living with DMD. This is a chance for your family to contribute to improving the care for all families with DMD across the U.S.
If are DMD patient or family member and are interested in participating in this survey, please contact Eric Graf, at the Neuromuscular Disease Research Center at 503-418-2124 or neuromus@ohsu.edu for more information.
