Cedars-Sinai Named Center of Excellence by Two Patient Advocacy Groups for Research and Care of Patients with Neuromuscular Disorders
Major Patient-Focused Advocacy Groups Recommend Cedars-Sinai for Those with Guillain-Barré Syndrome, Chronic Inflammatory Demyelinating Polyneuropathy and Charcot-Marie-Tooth Disease
Article ID: 620733
Released: 17-Jul-2014 5:00 PM EDT
Source Newsroom: Cedars-Sinai
Newswise — LOS ANGELES (July 17, 2014) – Cedars-Sinai has been named a center of excellence by two major advocacy groups for patients with neuromuscular disorders: Guillain-Barré Syndrome/Chronic Inflammatory Demyelinating Polyneuropathy Foundation International and the Charcot-Marie-Tooth Association.
“Patients with uncommon neuromuscular disorders often spend years searching for a correct diagnosis, as well as doctors who understand their symptoms and medical centers that can provide the most advanced testing and treatment,” said Richard Lewis, MD, director of the Electromyography Laboratory and co-director of the Neuromuscular Clinic in the Department of Neurology. “Being designated a center of excellence by these patient-focused organizations reflects the fact that Cedars-Sinai has assembled a highly experienced team of professionals with particular expertise in the diagnosis and treatment of these disorders. We also conduct leading-edge research to discover underlying causes of disease and give patients access to the latest emerging treatments through clinical trials.”
Cedars-Sinai is listed as one of 18 GBS/CIDP centers of excellence worldwide. The foundation website says, “Based on levels of expertise, available treatments, facilities and research capabilities, these are the medical centers that we can unequivocally recommend as ‘Centers of Excellence.’”
Guillain-Barré Syndrome is an acute inflammatory disorder of nerves outside the brain and spinal cord that can lead to paralysis. The disorder is due to an autoimmune reaction, typically triggered by a viral or bacterial infection. Despite severe weakness, most patients have a good recovery. Treatment during the acute attack with intravenous immunoglobulin or plasma exchange can be beneficial.
CIDP can be considered a chronic form of GBS. It also attacks the peripheral nerves and is believed to have an autoimmune basis, but symptom onset is gradual and the disease is progressive or relapsing, extending over several years. Without treatment, severe disability can occur, but there are a number of therapies that can be helpful.
Charcot-Marie-Tooth, the most common inherited neurological disorder, damages nerves that control muscles, primarily affecting the legs and feet. At least 79 genetic defects have been identified as causes of the disease, named for the three doctors who first identified it. Symptoms, severity and time of onset vary greatly, depending on the genes involved. High arches in the feet and increasing awkwardness are very common first clues, and although the disorder often is considered rare, it is relatively common, affecting one in every 2,500 people.
Cedars-Sinai's Charcot-Marie-Tooth/Hereditary Neuropathy Center is headed by Lewis; Robert H. Baloh, MD, PhD, director of Neuromuscular Medicine; and Glenn Pfeffer, MD, director of the Foot and Ankle Program at Cedars-Sinai’s Orthopaedic Center and co-director of the Cedars-Sinai/USC Glorya Kaufman Dance Medicine Center.
The CMT Association identifies 16 centers of excellence, with 13 in the U.S. and two in California. Cedars-Sinai is the only one in Southern California.
According to the website, “The CMTA has a mission to improve the quality of life for those with CMT. One way we accomplish this mission is by sponsoring patient-centric, multidisciplinary CMT clinics, staffed by some of the highest quality CMT clinicians and researchers in the world. Through these CMTA-designated Centers of Excellence, children, adults and families affected by CMT can be assured of receiving comprehensive care by a team of CMT experts. Each Center of Excellence has been vetted by the leaders of the CMTA’s Scientific Advisory Board …”
Cedars-Sinai’s center also is one of 15 worldwide research sites of the Inherited Neuropathies Consortium of the Rare Diseases Clinical Research Network. The consortium is funded by the CMTA, the Muscular Dystrophy Association and the National Institutes of Health. Worldwide, more than 7,000 patients with CMT have been enrolled in protocols during the past three years. Their data, de-identified to protect patient privacy, is housed in a common repository. As a result of this collaboration, more than 10 new gene disorders have been identified, a new CMT evaluation scale for children has been established, along with a new evaluation system for adults, and an infrastructure has been developed to perform natural history studies and clinical trials for CMT.
Cedars-Sinai also is designated as a center of excellence by the Hereditary Neuropathy Foundation, which supports those living with CMT.
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