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Synageva BioPharma is a clinical stage biopharmaceutical company dedicated to discovering, developing and delivering medicines for patients with certain rare disease and unmet medical needs. Their lead program is an investigational therapy for Lysosomal Acid Lipase (LAL) Deficiency, which has two presentations– an early onset form in infants (also known as Wolman disease) and a late onset form in children and adults (also known as Cholestryl Ester Storage Disease or CESD). The early onset form occurs more often in those of Persian or Persian Jewish descent.
They have designed a short 10 minute survey to determine the awareness of people in the Persian/Persian Jewish community of the increased risk of LAL Deficiency, HIBM, and other genetic conditions.
We would like to ask your assistance in taking this survey, and in sharing the survey link with your friends and relatives who are of Persian or Persian Jewish descent. They will be collecting survey responses for approximately one month, and donations will be made to LAL Solace, the patient organization devoted to LAL Deficiency, and ARM, the patient organization devoted to Hereditary Inclusion Body Myopathy (HIBM) in recognition of survey responses received.
Please click on the following link to access the survey; Survey Regarding Genetic Issues in Persian Jewish Community.
Thank you in advance for your assistance in participating in and sharing this survey!
Ultragenyx Pharmaceutical has announced that recruitment has begun for a Phase II Trial study for HIBM patients. Recruiting will soon begin at 3 locations (St. Louis, Los Angeles and New York) in the United States.
To be eligible, patients must be between 18 and 65 years of age, must have a documented diagnosis of GNE myopathy (HIBM), must be able to walk 20 meters independently (may use orthotics and assistive devices) and able to comply with all study procedures including fine needle muscle biopsies of the upper and lower extremities at Baseline at 24 and 48 weeks.
Click Ultragenyx - HIBM Sialic Acid Phase II Trial Recruitment for more info.
A Natural History Study of Patients with Hereditary Inclusion Body Myopathy (HIBM)
We are currently screening and recruiting patients for the “Natural History Study of Patients with Hereditary Inclusion Body Myopathy (HIBM)” at the NIH. The Natural History study is an initiative to collect appropriate data that is essential in preparation for treatment trials. The goal of this research study is to better understand the signs, symptoms, and clinical course of HIBM and to develop better tests to measure the progression of the disease.
By participating in this study, you will help to improve our understanding of HIBM, which is an important contribution to families and individuals affected by this condition. This study will provide information that will help shape subsequent investigations of potential treatments for HIBM.
Our timeline includes the initiation of the Natural History study for HIBM in September 2011 and the Clinical trial of ManNAc in HIBM no sooner than June 2012.
People eligible to participate in these studies will have travel expenses covered by the NIH, including those traveling form outside the US. Also, travel and lodging expenses may be paid for a companion, if medically indicated.
Visit their website! http://hibmstudy.nhgri.nih.gov
Here is another grassroots project only this one is happening in South Korea. Steve Kim, an accomplished automotive designer, decided to do his very first trialthlon to raise funds and awareness for HIBM. We love these grassroots project! Consider getting involved and start your own grassroots fundraising effort.
Check out Steve's project and read his story today! Thank you, Steve, for your involvement! http://www.crowdrise.com/trialthlonhibm
San Francisco to Santa Monica
Bike for Kam is back for its second annual grassroots fundraising efforts! Bike for Kam was started by HIBM patient, Kam, and her friends. It's about a group of friends riding a 7-day, 500 mile coastal bike tour from San Francisco to Santa Monica to raise funds and awareness for all those afflicted with HIBM. The B4K team completed their 500 mile trek on Saturday, May 19, 2012. Check out this amazing little local project doing not so little things. Currently, they are still taking donations. Watch their 2012 promo video below and consider donating and passing their project around! This is not an ARM funded event and 100% of the donations goes directly to HIBM medical research.
Donate and find more information at http://www.bikeforkam.com
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Read about ARM's upcoming telethon, how you can get involved by creating your very own grassroots fundraiser, patient corner featuring Rushabh's story, clinical trials and HRG's, ARM funded research lab, extradorinary work.
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ARM is now on Twitter. Get connected today and tell all your friends! http://twitter.com/hibmarm
ARM is on Facebook! Want to help, but don't know how? “Like” us on Facebook and don't forget to ask all your friends to like us, too! It's as easy as sending personal requests to your Facebook friends and encouraging them to become a fan of ARM.
When you “LIKE” us you are helping to spread awareness. The more fans, the more people will know about this rare condition; HIBM, and thus the more opportunities to continue treatment development. We need your friendship and your voice!
Middle-Eastern Genetic Panel - The people of Iran and Middle-East have higher risk for specific genetic disorders. We are currently offering a testing panel that will allow expecting parents to discover if their children are at risk. The tests can be done with a mouth swab, and there is no need for blood draw. Some or all of the tests may be covered by your insurance depending on your risks and your health insurance coverage. For ordering the tests, we can send the necessary information to your doctor. For more information, please contact HIBM Research Group laboratory at (818)789-1033
or visit: www.hibm.org/hrg
Dr. Babak Darvish's story, “Doctor determined to Cure Rare Disease”, was aired Friday, July 24 Jefferson on Channel ABC7 (Los Angeles).
Watch the Video Clip HERE
LOS ANGELES (KABC) – Throughout the year ABC7 recognizes individuals making a difference in their communities.
The Jefferson Awards is an award organization co-founded by Jacqueline Kennedy-Onassis in 1972 to encourage and honor people for their contributions through community service. It is an organization based on a simple idea – the belief that one person can make a difference.
To read more about the Jefferson Award or to read the heartfelt nomination letter written by Dr. Darvish's colleague, Crystal, please click here.
Dr. Babak Darvish, MD,Co-founder of ARM, and Kam Redlawsk were featured on ABC7's Health Living by reporter, Denise Dador on Thursday, August 20, 2009.
Kam Redlawsk, an HIBM patient, went through years of searching for a diagnosis for her unknown debilitating condition. By chance her visit to California would lead her to ARM and thus the Darvish Brothers, Co-founders of ARM, who had set out 10 years ago with the mission to cure HIBM.
Using her Industrial Design background she created ARM's new look and patient campaign in hopes to gain more awareness towards a debilitating disease that has no cure, but is cureable. She continues as ARM's pro-bono Creative Director and patient advocate.
Join ARM, the Darvish Brothers, MD, Kam and all HIBM patients by giving them a voice.
Watch ABC7's News story HERE
Read about more patients like Kam at: click here.
The result of scientific study on a mouse model gives hope that N-Acetylmannosamine, a sugar-like molecule, may help HIBM patients. See the news release and original scientific article published on June 1, 2007 issue of JCI. The study used a mouse model produced by HIBM Research Group (HRG), an ARM funded laboratory.
For more information and published findings
The result of the IVIg therapy, the first clinical therapeutic trial on HIBM, is now published. You may download the full text of the article at BMC Neurology. The study was made possible by patients referred from HIBM Research Group ( HRG), an ARM funded laboratory.