HIBM Research Group (HRG) is actively involved in advancing research and clinical trials for GNE Myopathy, a rare genetic disorder. The organization sponsors various clinical trials, including studies on N-Acetylmannosamine (ManNAc), gene therapy, and stem cell therapy, aimed at understanding and potentially treating the disease. The trials utilize mouse models to explore the efficacy of these treatments, with promising results indicating that ManNAc may offer hope for patients. Additionally, HRG collaborates with institutions like the National Institutes of Health (NIH) to conduct natural history studies that gather genetic and medical data from patients, which is crucial for developing future therapies.

The organization also emphasizes the importance of genetic testing for patients diagnosed with HIBM, as it is essential for participation in clinical trials. HRG has been instrumental in facilitating access to genetic testing and has supported various research projects through grants and funding. Their commitment to patient advocacy is evident in their efforts to empower individuals affected by HIBM, providing them with knowledge and resources to navigate their condition. Overall, HRG's multifaceted approach combines research, clinical trials, and patient support to drive progress toward effective treatments for GNE Myopathy.

United States

HIBM (Hereditory Inclusion Body Myopathy) is a progressive and debilitating muscle wasting disorder caused by a gene defect. It touches those betwen the ages of 20 to 40 and, although progression is slow, it typically leads to total disability within 10-15 years. HIBM Research Group is laboratory funded byARM (the Advancement of Research for Myopathies,a leading supporter in research toward the cure).

HIBM Research Group (HRG)

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