HNF’s website provides CMT community, academia, and biotech industry a hub for exploring research, clinical trials, vital resources, and ways to get involved.
NEW YORK , NEW YORK , UNITED STATES , February 8, 2023/EINPresswire.com/ — – Charcot-Marie-Tooth (CMT) disease affects 1:3300, nearly 3 million people worldwide, and many remain undiagnosed.
– The Hereditary Neuropathy Foundation has redesigned its website to provide the CMT community, academia, and biotech industry a user-friendly hub for exploring current research, clinical trials, vital resources, and ways to get involved. In addition, it offers translation from English into eight languages.
The Hereditary Neuropathy Foundation’s (HNF) mission is to increase awareness and accurate diagnosis of Charcot-Marie-Tooth (CMT) and related inherited neuropathies (IN), support the CMT community with critical information to improve quality of life, and fund research that will lead to treatments and cures. Today it launched an exciting and user-friendly website focusing on growing global interest in research and therapy development for CMT. Interest in such work has grown since HNF hosted its Externally-led Patient-Focused Drug Development Meeting for the FDA in 2018, followed by the publication of the “Voice of the Patient” report. This groundbreaking meeting identified the lack of understanding of CMT and the subsequent barriers to treatments, such as; access to genetic testing and patient registry data, poor clinical trial design, and identification of symptoms that matter most to patients. Since then, HNF has been ultra-focused on eliminating these barriers. The well-designed and functional website provides the latest updates on research, clinical trials, and vital patient resources – including the CMT Genie for diagnosis and the Global Registry for Inherited Neuropathies (GRIN) patient registry. Valuable patient data is collected in the enhanced GRIN registry platform which was made possible with a large contribution by Brian and Katie McCormack, Chicago-based investors and philanthropists.
“I am happy to support this patient registry which now includes research regarding comorbidities with the NIH-funded ClinGen surveys. I believe this will lead to a better understanding of CMT – the way it impacts families dealing with this disease – and prioritize treatments for kids prior to any significant deterioration.”
On March 1, 2023, at 7:00pm ET, HNF will host a webinar, “Making Sense & Science of CMT Symptoms.” The webinar will provide resources for various CMT-related symptoms and highlight CMT patients’ key role in accelerating important research. Without patient participation, researchers won’t have the essential patient information to develop drugs, gene therapies, and clinical trials for CMT and IN. HNF will present how the CMT community can turn their symptoms into science, enabling better clinical trial design.