In order to advance research to improve medical treatment and care for those living with Inherited Neuropathies (IN), such as Charcot-Marie-Tooth (CMT), the Hereditary Neuropathy Foundation (HNF) is relaunching a more dynamic and user-friendly online survey.
The HNF will host a Patient-Centered CMT Behavioral Health Summit on Sept. 28 and 29 at the College Park Marriott Hotel and Conference Center in Hyattsville, Maryland. To register and for more information, go here.
The new survey-based study, called the Global Registry for Inherited Neuropathies (GRIN), is designed to foster a better partnership between IN patients and researchers with the goal of accelerating treatment discoveries. Other than physical and occupational therapy, and medicine for pain, there are no standard treatments for heredity neuropathies.
The survey is meant to collect key information from a diverse pool of patients to give scientists, pharmaceutical companies, insurance companies and the Federal Drug Administration insight into the lives and genetics of people with IN. GRIN will also serve as a patient recruitment source for clinical trials.
The hope is that tens of thousands of patients globally will participate in GRIN, open to those 18 and older. The first four study categories are: natural history study, respiratory dysfunction, medical cannabis, and voice-activated surveys on the disease’s impact.
“By incorporating patient perspectives, we are enabling an enriched understanding of the experience of living with CMT,” Allison Moore, founder and chief executive officer of HNF, said in a news release.
“This in turn is a robust tool to de-risk research investment by industry and to facilitate systematic research and therapy development to improve patient outcomes and endpoints. GRIN has the immense potential to accelerate and improve therapy development.”
Hereditary motor or sensory neuropathies include inherited nerve disorders affecting the peripheral nervous system, the most common type being CMT, affecting about 150,000 people in the United States.
IN symptoms vary depending on the type, and may include numbness, tingling and pain in the feet and hands. They also may include weakness and muscle loss, particularly in lower legs and feet. Some people may have scoliosis, thin calf muscles, and foot and hand deformities.
Organizers said the health summit, which will focus on behavior and emotional health, will provide an important CMT toolbox that patients can apply to their daily lives for improved fulfillment and wellness.
“We often hear so much in regard to the physical aspects of disease and disability but very little on how it impacts our mental well-being, which for many can be more devastating,” said Estela Lugo, HNF medical outreach manager.
The summit will include an interactive empowerment session called “Fuel Your Confidence” designed by thought leader Lisa McCarthy, co-founder of the Fast Forward Group, which conducts leadership programs.
Orthopedic surgeon Glenn B. Pfeffer, who specializes in CMT procedures, will discuss how surgery outcomes can affect behavioral health. A workshop on Assertiveness and CMT will be led by Lainie Ishbia, founder of Trend-ABLE, a website designed to support women with invisible disabilities. Registered dietician and holistic health coach Alana Kessler will moderate Nutrition and CMT.
The nonprofit HNF aims to increase awareness and proper diagnoses of CMT and related inherited neuropathies as well as to support patients and their families through education. It also funds research it hopes will lead to treatments and cures.
It also led the Therapeutic Research in Accelerated Discovery (TRIAD) program, a collaborative effort with academia, government and industry, to develop CMT treatments.