Beneficiary - About HIBM and ARM
HIBM (Hereditary Inclusion Body Myopathy, AKA GNE Myopathy) is a rare genetic disorder that causes progressive muscle wasting and weakness. The weakness and severity can vary from person to person. In some, weakness in the legs is noticed first. In others, the hands are weakened more rapidly in the legs, but in all cases HIBM has the potential to take those it touches to complete quadriplegia within a timespan of 10-15+ years, confining many patients to a wheelchair during the most productive years of a person's life.
Muscle degeneration usually starts around the ages of 20-30 although science has seen onset as young as 17. Patients lose muscle strength every day until they can no longer walk, use theirs arms, hands, fingers or even hold their head up. This is happening to Kam and to many patients. Despite HIBM's rarity (approx 1000 patients worldwide) patients can be found in every corner of the world, affecting many different ethnicities and backgrounds from Japanese, Jewish Persian, Irish, Indian, Caucasian, Korean, Chinese, Italian and more.
Because of the extreme rarity HIBM is known as an 'Orphan Disease'. According to US criteria, an orphan disease is one that affects fewer than 200,000 people and yet there are more than 5,000 such rare disorders worldwide. Because of it's orphan disease status it is more difficult to be "adopted" by the pharmaceutical industry because it provides little financial incentive for the private sector to make and market new medications to treat or prevent it because it is not labeled as "high profit".
The good news is that through grassroot efforts the technology exists to treat/cure HIBM and halt its progression. One of the treatments is already being tested on humans, and a gene therapy technology that ARM created has been FDA greenlit for human clinical trials. The human clinical trials could start TODAY if they had the necessary funding.
About ARM (Advancement of Research for Myopathies)
Fifteen years ago two siblings noticed a disabling condition beginning to express in their bodies. It was HIBM. At the time they were medical students. Realizing HIBM could be treated they set forth to help fund and find a cure. Together, brothers Dr Daniel Darvish and Dr. Babak Darvish founded ARM; a non profit organization dedicated to finding a cure and increasing HIBM awareness. Because of the brothers' research great strides have been made in the research worldwide and today technology exists to cure and or treat HIBM.
The primary bottleneck has always been lack of funding and lack of awareness.
Dr. Babak Darvish serves as the ARM President and Dr. Daniel Darvish founded HRG (HIBM Research Group); a nonprofit biomedical research lab based in California. HRG is the ONLY research lab in the world that dedicates itself full time to HIBM research and Dr. Daniel Darvish has dedicated his life probono to further HIBM research.
In the short span of 15 years ARM/HRG has:
- funded vital research around the world (Israel, Japan, Canada, Texas, Los Angeles…)
- located the mutated gene responsible for HIBM
- proposed the potential cause of muscle wasting: lack of sialic acid
- improved tests to measure sialic acid in HIBM patients
- formulated the first therapeutic hypothesis including, but not limited to, substance therapy (ManNAc, Sialic), gene therapy and stem cell
- developed and offered streamline DNA testing for HIBM which led to faster and accurate diagnosis
- development of FIRST HIBM mouse model and the sharing of other critical biomaterials and reagents to researchers worldwide
- introduced simple swab test for HIBM and innovated and developed a remarkably elegant and HIBM test with results in 1-2 days for the Middle Eastern mutation of GNE
- closely work with NIH and FDA as a respected colleague
- advancement of work on gene therapy vector, opening the way for a rapid and streamline drug development pathway
Today two potential therapies exist because of ARM. Sialic acid which is currently undergoing human trials with FDA and Gene Therapy which has been approved by FDA to undergo human trials in California but needs 5 million to begin.
Only with your support and donations can ARM/HRG take all his remarkable research and development to the finish line. It is no longer about finding the cure; it is about funding the cure and seeing it though human trials and ultimately making it available to patients. Every little bit helps and so can you.
For more info about ARM visit: www.hibm.org
*ARM is an accredited 501(c)(3) non profit. All donations are tax deductible. EIN#95-4837946