Newsletter July 7, 2016  

What Caught Our Eye

Of Mice and Men…and Maybe Muscle
By Kenneth Schikler, MD

Editor’s Note: Engage in the conversation about this topic with the authors on the What Caught Our Eye blog.


As I am sure occasionally happens to us periodically, a young toddler was referred to me by gastroenterology after being evaluated for hepatopathy regarding elevated “liver enzymes.” This young boy ended up having muscle disease, and genetic testing confirmed Duchenne Dystrophinopathy. While he has been sent on to our MDA clinic for his future care, I was interested in looking into what was new in Muscular Dystrophy. In the Proceedings of the National Academy of Sciences (vol 112, no 41, pp 12864-69), Whitehead et al report on “A new therapeutic effect of simvastatin revealed by functional improvement in muscular dystrophy.”

In both Duchenne dystrophy and dystrophin-deficient mice, muscle degeneration involves chronic inflammation, oxidative stress, and fibrosis. Despite concern about statin-induced muscle disease in humans (<1% compared to placebo), they point out that statins have been associated with improved skeletal muscle health in in ischemic limb disease and diminished oxidative stress and inflammatory cell infiltrate.

Read more...

CARRA News & Announcements

Please vote in the runoff election for CARRA Small Centers Vice Chair!

The initial voting period for the Small Centers Vice Chair position has ended. With no single candidate achieving over 50% of the vote, we are holding a runoff election between the top two candidates:

        Theresa Wampler Muskardin, MD        
 Simona Nativ, MD

We need voting members from Small Centers to vote again! We know you may have previously voted, but we appreciate the extra effort. Email ballots were sent out via SurveyMonkey from [email protected]) on June 28th. If you did not receive your ballot, please click here* to participate in this important runoff election by Sunday, July 10th at 8:00 PM EST, 5:00 PM PST. Review the candidates’ personal statements and biographical sketches in the voting guide prior to starting the election survey. We really need your active participation in the voting process. As a member of CARRA, voting in elections is a responsibility! You will be asked to provide your name so that we can track members’ participation. All votes remain confidential. Thank you for taking the time to vote. We know you have lots of competing priorities and appreciate your support of CARRA.

Grants and Funding

CARRA grants
CARRA offers members various funding opportunities to further research that leverages the CARRA network and positively impacts the pediatric rheumatology scientific community and advances the CARRA mission. Additional information for the different grant opportunities, including the application instructions and templates, is available on the CARRA website.

Cure JM Foundation 2016 Funding Opportunity
The Cure JM Foundation will fund research or educational awards in two different cycles in 2016. The vision of Cure JM includes increasing awareness of juvenile myositis and funding research in juvenile myositis that will ultimately lead to a cure. Proposals will be accepted through August 31st, 2016. Each cycle will fund a maximum of three different proposals for up to $50,000 each. Read more…


Registry Update
Registry Enrollment: 1,421
STOP-JIA Enrollment: 43

Registry Stats
Enrollment: 1,421
11 sites have enrolled 5 or fewer patients
30 sites have enrolled 6-49 patients
7 sites have enrolled 50 or more patients

Top Registry Sites:
PI Sarah Ringold/SC Luke Reichley, Seattle Children's Hospital: 176

PI Christi Inman/SC Suzy Jones, University of Utah Hospitals: 144

PI Jennifer Weiss/SC Mary Ellen Riordan, Hackensack UMC: 137

STOP-JIA

STOP-JIA Stats:
Enrollment: 43
7 sites have enrolled 1 patient
7 sites have enrolled 2-4 patients
3 sites have enrolled 5 or more patients

Leading STOP-JIA enrollers
PI Sarah Ringold/SC Luke Reichley, Seattle Children's Hospital: 8

PI Mara Becker/SC Chelsey Smith, Children’s Mercy Hospital: 5

PI Jennifer Weiss/SC Mary Ellen Riordan, Hackensack UMC: 5

Our June goal for STOP-JIA is 54 enrolled patients! For subsequent months, enrollment needs to be 20 subjects/month to stay on target.  Please do all you can to enroll at least 2 patients a month! 
Read more...

Early Investigators

The Early Investigator group is launching an Aims Page Review program in which Early Investigators can get feedback on the Specific Aims page of their grants from CARRA members who have experience as grant reviewers. The Specific Aims page is considered the most important part of any grant and grant writing success depends on making it as good as possible. If you are submitting a grant in the near future and are interested in getting constructive feedback, please contact Committee Chair, Jay Mehta.

Research Spotlight

CARRA members Ginger Janow, Laura Schanberg, Betsy Mellins, Yuki Kimura, Soko Setoguchi, Ray Schneider, along with Rayfel Schneider and Vic Hasselblad, writing for the CARRA Legacy Registry Investigators, are the authors of a manuscript examining the s-JIA cohort of the CARRA registry between 2010 and 2013, "The Systemic Juvenile Idiopathic Arthritis Cohort of the Childhood Arthritis and Rheumatology Research Alliance Registry: 2010–2013."

First author Ginger Janow, MD, commented, “An important take-away from the paper is that our systemic JIA patients are doing well overall with low morbidity.  However, pockets of more severe disease appear to exist, particularly amongst African American patients and those whose arthritis persists even though their systemic symptoms have resolved.” 

The authors conclude that future research will need to assess these groups more closely.  Practice patterns in the choice of biologic agents appeared to change during the study period. There appeared to be a shift away from TNF-alpha inhibitor use and an increase in the use of IL-1 and IL-6 inhibitors. The new CARRA Registry is collecting more detailed information such as medication doses (specifically steroids) and start/stop dates to further define trends in treatment and outcomes.

Arthritis Foundation News

2016 National JA Conference
The Arthritis Foundation’s annual Juvenile Arthritis Conference is where children, teens, young adults and their families come together from across the world to gain knowledge about juvenile arthritis and other childhood rheumatic diseases. Jason Jones, CARRA’s Senior Director of Research, and Kelly Mieszkalski, CARRA’s Executive Director, will host a CARRA exhibit at the JA Conferences.  If you are planning to attend, please plan to stop by the CARRA exhibit.

Local Connections
The Arthritis Foundation has camps and family events across the country that provide kids and teens and their families the opportunity to network, learn and play with others in their local community.

Local Leadership Board
In communities nationwide, the Arthritis Foundation is working with local volunteers to build Leadership Boards to achieve our goals in fundraising, volunteer engagement, advocacy, juvenile arthritis and other pediatric rheumatic diseases, and help and support. For more information on how you can get involved, please contact Alison Imamura, [email protected].

Read more…

Connections

Save the date! 2nd Global Conference on Myositis, May 5-8, 2017
The conference will take place at the William F. Bolger Center in Potomac, MD. This year's event will feature interdisciplinary scientific presentations of state-of-the-art research in all areas of myositis, with a focus on trainees and young investigators and input from myositis patient support group leaders. The abstract submission deadline is December 16 (http://www.gcom-int.com/abstracts.html).  For the early registration discount, click https://www.regonline.com/builder/site/Default.aspx?EventID=1839144

Scleroderma Foundation National Patient Education Conference
Scleroderma Foundation announces the National Patient Education Conference in New Orleans, July 29-31. Any child, a patient or a sibling of a patient, is welcome to join the program. If the child has received a scholarship in the last three years, he/she is not eligible for another scholarship at this time. There will be programming for children, as well as workshops geared toward adults who have a juvenile patient in their lives. For more information on the conference please call Ryan Burrill at 800-722-HOPE (4673) or email him at [email protected]. Visit the conference website at www.scleroderma.org/conference.

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