The commitment to a cure continues… ARM's annual telethon will be broadcasted LIVE on SUNDAY, DECEMBER 4th, 2011 TIME: 12-7 PM STATIONS: TimeTV, Highvision, ParsTV, TiTV and Channel 18 (ksci)

It's no longer about finding a cure; it's about funding a cure! Help us make 2011 Telethon the best yet!

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 our website! http://hibmstudy.nhgri.nih.gov

Thank you to all the patients who have decided to participate in the exciting Phase I trial for Sialic Acid! This first study is a short study to see how the investigational drug circulates in the body.

Currently, half of the recruitment patient quota has been filled in both the New York and California clinical trial locations and Ultragenyx is looking for a few more patients to enroll, but space is limited. The sooner Phase I trial is completed, the sooner Ultragenyx can move forward to Phase II and Phase III Trials, but this cannot happen until complete enrollment has been achieved for Phase I Trial.

1) Travel expenses will be covered by Ultragenyx.
2) If you want to participate, but don't have an attending physician or a Neuromuscular Specialist: You may contact us and we can provide information to you and your family doctor and refer you to an MDA clinic closest to your home.
3) To be eligible, patients need to have their HIBM diagnosis confirmed with a genetic test. Ultragenyx pays for your genetic testing Genetic testing will be important for Phase II and Phase III Trial.

To obtain testing at NO COST, patients should contact their doctor and ask them to order sample collection kits from Ultragenyx for testing.

* * * *
Contacts:
To order test collection kit:
John Ditton, jditton@ultragenyx.com

To learn more about the Clinical Trials:
Mari Maurer, RN, BSN, mmaurer@ultragenyx.com

Phase 1 Trial Site Contacts/Locations:

Clinilabs, Inc.
212-994-4567
423 W. 55th Street, 4th Floor
New York, NY 10019
participate@clinilabs.com
www.clinilabs.com

West Coast Clinical Trials
714-668-1500 ext 4009
3545 Howard Way, Suite 100
Costa Mesa, Ca. 92626
ptinfo@wcct.com
www.wcct.com

Once again, thank you to all the current participants.

ARM is now on Twitter. Get connected today and tell all your friends! http://twitter.com/hibmarm

www.facebook.com/curehibm

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!

Ultragenyx Pharmaceutical Announces the Start of a Phase 1 Clinical Trial for Hereditary Inclusion Body Myopathy (HIBM) - also known as Nonaka Myopathy or Distal Myopathy with Rimmed Vacuoles (DMRV)

(Novato, CA, July 20, 2011) – Ultragenyx Pharmaceutical has announced that recruitment has begun for a first clinical study for HIBM patients. To be eligible, patients need to have their HIBM diagnosis confirmed with a genetic test. This first study is a short study to see how the investigational drug circulates in the body.

“People with this rare disorder previously had no compelling reason to undergo genetic testing to understand their genetic mutation or to obtain a definitive diagnosis,” said Emil Kakkis, MD, PhD, President and CEO of Ultragenyx. “As clinical trials are initiated patients will need a documented diagnosis of HIBM to be eligible, so we are helping them to get genetic testing.”

To obtain testing at no cost, patients should contact their doctor and ask them to order sample collection kits from Ultragenyx for testing. * * * *

Contacts:
To order test collection kit; John Ditton
jditton@ultragenyx.com
To learn more about the Clinical Trials; Mari Maurer, RN, BSN
mmaurer@ultragenyx.com

Phase 1 Trial Site Contacts:

Clinilabs, Inc.
212-994-4567
423 W. 55th Street, 4th Floor
New York, NY 10019
participate@clinilabs.com
www.clinilabs.com

West Coast Clinical Trials
714-668-1500 ext 4009
3545 Howard Way, Suite 100
Costa Mesa, Ca. 92626
ptinfo@wcct.com
www.wcct.com

Ultragenyx PDF Press Release
ux001_cl101_phase_1_online_ad_w_irb_changes_2011jul20_2_.pdf

Track the 'Bike for Kam' team epic ride cross country in their online daily log. Read their 500 mile daily stories at: www.bikeforkam.com

Ending their 500 mile trek the team will be arriving at Santa Monica Pier on Saturday, April 29, 2011. Come celebrate the team, donors and those 500 miles they biked at a Sunday 'Bike for Kam' After Party! We know, it’s a Sunday, but the guys just biked 500 miles fully loaded. They deserve a pat on the back and they would love to see you. Tell all your friends and pass it on.

Thanks to all who donated and supported 'Bike for Kam'! Currently, at $16,422; 82% of their monetary goal, donation lines will stay open for the next week or so. If you haven't donated you still can and help them reach their 20K goal!

Brennan’s Pub, home of turtle racing
Sunday, May 1 @ 7pm
Brennan’s near Venice, CA
4089 Lincoln Blvd
Marina del Rey, California 90292

Sunday, April 24, 2011 the Bike for Kam Team departed San Francisco's Golden Gate Bridge at 6:15 PM. They have been in regular contact with Kam and she is logging their trip in an online daily bike journal @ www.bikeforkam.com. Because of you the team has reached 75% of their 20K fundraising goal and needs you to help them to the finish line. Follow their 500 miles through pictures and stories and support them by donating @ www.bikeforkam.com

For the DAY ONE post a short video was put together to chronicle the weekend preparations, team arrivals in San Francisco and departure from Golden Gate.

* 100% of proceeds from Bike for Kam goes directly to HIBM medical research.

Bike for Fun . Bike for Hope

Bike for Kam is a grassroots project about a group of friends coming together to complete a coastal bike tour from San Francisco to Santa Monica (Los Angeles) on April 25 to April 29, 2011. The purpose of their journey is to raise funds and awareness for their friend Kam, and for all those afflicted with HIBM (Hereditary Inclusion Body Myopathy). Through this project we hope to raise awareness of HIBM, so that we may soon find a cure for this rare condition.

As a grassroots project, all the work has been voluntarily created and expensed by the team above.

Please VISIT BIKEFORKAM.com TODAY! Donate and Pass it on to all your friends and family. Remember, no donation is too small.

For More Information on the project, read about Kam and the team or to Donate, visit:
http://www.bikeforkam.com

100% of Proceeds goes towards HIBM Medical Research.

A very special thanks to the Running for Cara team who raised a grand total of $18,143 in October of 2010! If you have always wanted to help, but don't know how, organizing a grassroots project in your community is a great way to get involved!!

Richard, Vince and Josh has joined crowdrise to help their dear friend, Cara, who is afflicted with HIBM. In an effort to raise awareness and funds towards the research for HIBM (Hereditary Inclusion Body Myopathy), they will be running for Cara in a marathon (40,000 steps) in Washington DC on October 31, 2010.

“When I first met Cara, at the coffee shop in our university, I didn’t ask her why she walked with a cane and a limp…My reaction was one of confusion and fear- terrified that things would progress, uncertain for my new friend’s future…It was terrifying to see a vibrant, young person affected by this terrible disorder in the prime of her life…I no longer take every step for granted. I’m running for Cara - a marathon, something I swore to never do again- to help raise money to fund the cure for HIBM. Perhaps my dear friend, and others with HIBM, can one day run one with us; afterwards we’ll laugh and complain about the “pain” in our feet together; knowing that we’re blessed to have it.”

Please read more and visit his fundraising page at:
http://www.crowdrise.com/CUREHIBM-running4cara

Read Cara's story at:
http://www.hibm.org/arm/community:cara_yar_khan:cara_yar_khan

ARM's EIN #: 95-4837946

If you've always wanted to help ARM, but just didn't know where to start–here is your chance! Start a grassroots fundraising page at crowdrise.com and help us get closer to the cure for this very rare and debilitating muscle condition.

ARM has just joined CROWDRISE.com, an online social fundraising, online giving and volunteer community. Crowdrise is about volunteering, raising money for Charity and whether you're running a marathon, volunteering, want to use your birthday to raise money, concert; you can create your own Fundraising Page on Crowdrise and choose 'Advancement of Research for Myopathies'; to raise money for from wherever you are and whatever fundraising idea you can dream up!

http://www.crowdrise.com/CUREHIBM
EIN #: 95-4837946

If you need help and have questions, please contact Kam at kredlawsk@hibm.org

ARM's Quarterly (September) Newsletter is available for download and viewing. This celebratory 10th Anniversary newsletter tells the story, where we came from and where we are today. Click icon below for download.

* 2010-09

To view past Newsletters please click here.

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

July 22, 09

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.

Rare Disease Day is a campaign dedicated to increasing awareness with policy makers and the public of rare diseases and of their impact on patients’ lives. Rare Diseases are chronic, progressive, debilitating, disabling, severe and often life-threatening.

The hope is that every year on February 28 Rare Disease day will increase awareness of rare diseases, the special challenges encountered by those affected, and the need for research to develop safe, effective treatments or cures. The rare disease patient is the orphan of most health systems often without diagnosis, without treatment and without research.

( For more info: rarediseaseday.org)

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.