Mission & SummaryMission: HRG was organized to speed up development of effective treatments for Autosomal Recessive Inclusion Body Myopathy (IBM2). Our mission includes accomplishing this goal in the most efficient manner possible, with special emphasis on considerations of results/duration/cost value of biomedical research activities.Summary: Hereditary Inclusion Body Myopathies (HIBM or h-IBM) are a diverse group of muscle wasting disorders that share similar histopathology with sporadic Inclusion Body Myositis (s-IBM) and senile plaques seen in Alzheimer's brain disease. Although histologically similar, various forms of HIBM are genetically and clinically diverse. The autosomal recessive form, IBM2, is the most common. It usually affects young adults, between the age of 20-35 years, and often leads to severe disability and confinement to a wheelchair in 10-15 years. Although worldwide it is an "orphan" disease, IBM2 is the most prevalent genetic disorder amongst people of Iranian-Jewish heritage, accounting for a carrier rate of 1-2 in 20 persons, and an incidence rate of 1-2 in 1000. IBM2 is a recessive genetic disorder, which means it can happen to anyone without any warning. More than 98% of the patients have healthy parents, who were carriers of the disease without knowing. More than 85% of the patients and their families had never heard of IBM2 prior to their devastating diagnosis. In the medical literature, IBM2 is also known as Quadriceps Sparing Myopathy (QSM), Distal Myopathy with Rimmed Vacuoles (DMRV), or Nonaka's Myopathy. The major goals of the organization is divided to 1) further clarifying the mechanism that leads to IBM2 muscle damage, 2) prevention of muscle damage cause by IBM2, and 3) muscle regeneration. Even though HRG personnel often make significant personal sacrifices in the pursuit of these noble goals, developing an effective treatment for a currently untreatable disorder can be very difficult and costly. Fortunately, recent scientific data suggest that IBM2 may be easier to treat than more common muscle wasting disorders, and the prospect of developing an effective treatment for IBM2 is very promising. |
 |