One minute, one dollar, pass it on.
We're asking for two things: give $1 and pass this email onto people you know. We're trying to raise $1,000,000 in 3 months to enable human trials of a promising sugar-like molecule to fight a debilitating disease. In the Internet age, reaching one million people in that amount of time is surely do-able.
HIBM (Hereditary Inclusion Body Myopathy) is a progressive and debilitating muscle-wasting disorder caused by a gene defect. It touches those between the ages of 20 and 40 and, although progression is slow, it typically leads to total disability within 10-15 years. Clinical trials are needed to test an exciting new treatment - they're scheduled and ready to go. We just need the money to fund them.
HIBM (Hereditary Inclusion Body Myopathies) are a group of rare/orphan genetic disorders. HIBM causes progressive muscle wasting and weakness that begins in young adulthood and can lead to very severe disability within 10 - 20 years. Besides clusters of patients amongst people of Middle Eastern, Jewish, or Japanese heritage, patients can be found in every corner of the world. HIBM has been identified in people Asian (Japanese, Korean, Chinese and others), European, South American, African, and Middle Eastern (Muslim, Palestinian, Jewish, Iranian) origins.
HIBM is also known as Distal Myopathy with Rimmed Vacuoles (DMRV), Quadriceps Sparing Myopathy (QSM), or GNE related muscle disease. It causes progressive muscle weakness and wasting. Muscle wasting usually starts around the age of 20 - 30 years, although we have seen young onset at 17 and old onset at 52. As such, it affects the most productive times of a person's life. It can lead to severe disability within 10 - 15 years, confining many patients to a wheelchair. The weakness and severity can vary from person to person. In some, weakness in the legs is noticed first. In few others, the hands are weakened more rapidly than the legs. Weakness is progressive, which means the muscle become weaker over time. HIBM does not seem to affect the brain, internal organs or sensation. The quadriceps are relatively spared, and remain strong until the late stages of disease, which is the reason HIBM is often referred to as Quadriceps Sparing Myopathy (QSM). It is caused by genetic variations in a gene known as GNE.
ARM (Advancement of research for Myopathies) is a 501(c)(3) non-profit organization with the primary goal of speeding up bio-medical research on IBM2, the Autosomal Recessive form of Hereditary Inclusion Body Myopathies (HIBM).
Founded by HIBM patients in 2000, ARM's mission is to support and inform patients and their families, to raise funds for research, to encourage researchers to study this rare disorder and to ultimately find a cure for Hereditary Inclusion Body Myopathy (HIBM). ARM, a non-profit organization, is the leading source of information, hope and knowledge for HIBM patients worldwide, and is the only non-profit organization specialized in this rare disorder. ARM offers scientific grants to research centers who are interested in working towards developing a treatment for HIBM.
Every year unsuspecting young adults are diagnosed with Hereditary Inclusion Body Myopathy (HIBM), a rare muscle wasting disorder for which there is no treatment or cure yet available. For them and all HIBM patients, ARM offers a ray of hope with research funding, support and an expanding campaign of public awareness. Only with your help can ARM continue its efforts to cure this debilitating disease.
ARM's purpose and goal is to accelerate biomedical research aimed at developing treatments for HIBM (recessive HIBM - Hereditary Inclusion Body Myopathy, Mendelian Inheritance in Man Catalogue # 600737), and skeletal muscle regeneration. ARM's mission is to accomplish this goal in the most efficient manner possible, with special emphasis on considerations of results/duration/cost value of proposed research.
ARM's vision is to have an effective preventive treatment for HIBM available for all current and future patients in the next few years, to offer regenerative treatments for patients who have lost muscle tissue, and to empower patients, and community, worldwide with knowledge and support.
For More information visit ARM's main website.
100% of Dollar Campaign donations will be applied towards reaching clinical trials. The funds raised will be used for completing needed toxicology studies based on FDA guidelines and regulations. The toxicology studies must be done prior to commencing clinical trials (testing in human patients) using a very promising sugar-like compound known as N-Acetylmannosamine (ManNAc). Any remaining funds will contribute towards gene therapy and stem cell therapy trials.