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A Publication of the Facioscapulohumeral Muscular Dystrophy Society WINTER 2014 FSH Watch CONNECTING THE COMMUNITY OF PATIENTS, FAMILIES, CLINICIANS AND INVESTIGATORS ADVOCACY CELEBRITIES JOIN OUR CAUSE Meet the FSH Society’s Honorary Board by JUNE KINOSHITA FSH Society, Lexington, Massachusetts ndy Warhol predicted that “In Athe future, everyone will be world- famous for 15 minutes.” In the era of reality television and viral videos, his words strike an eerily prescient chord. But it seems that FSHD is still waiting for its moment in MUHLRAD PAUL PHOTO: the limelight. It’s a disease that most of the Jeff Miller and Peter Jones chat at the FSH Society’s International Research Consortium meeting. public has never heard of. So why not harness celebrity culture to RESEARCH help raise the visibility of FSHD? Working with retired Hollywood agent and nation- ally known event consultant David Mirisch, I nternational Research Consortium Meeting the FSH Society reached out to figures from television, film, sports, fashion, and music Researchers gather on MIT campus and secured 15 members for our newly formed Honorary Board. by JUNE KINOSHITA These distinguished individuals include FSH Society actors from beloved films and television his past October 21-22, nearly a chair of the FSH Society’s Scientific Advisory . continued on page 14 Thundred researchers from around Board, began the meeting on a somber the world gathered at the David H. Koch note by paying tribute to MIT police officer Center for Integrative Cancer Research on Sean Collier, who was slain in April only the campus of the Massachusetts Institute steps from the Koch Center by the Boston of Technology for the annual FSH Society Marathon bombing suspects. Housman International Research Consortium said, “Many of us knew him, and it was a meeting. Located at the world epicenter very direct and personal experience. We are of biomedical research (forgive us for our dedicating our publications this year to Sean.” ICHARD WRIGHT Cambridge-centric perspective), there Around a dozen of the scientists and R was a palpable feeling of FSHD research observers attending the conference were PHOTO: PHOTO: having “arrived” in the big time. personally affected by FSHD. They included Max Adler Conference host David Housman, PhD, members of the FSH Society’s “virtual journal club” of scientist-patients who have WHAT’S INSIDE been meeting online to discuss current FSHD research. page 3 page 5 page 16 page 20 Baziel van Engelen, MD PhD, of the 2013H FS UMass Med School Champions Meet Raising Nijmegen Medical Centre in the Netherlands, Society Pioneer Launches FSHD in Cambridge, Awareness . continued on page 11 Award Research Center Massachusetts Through Football LETTER FROM THE EXECUTIVE DIRECTOR Looking Ahead Hope and concern for what 2014 brings Board of Directors Daniel P. Perez, Dear Friends, We go into 2014 with great hope, but also with deep President & CEO As we embark upon the concern for the challenges ahead. At the NIH, pay- William R. Lewis Sr., MD, Chairman New Year, my heart is lines have dipped below the 10th percentile, meaning Howard Chabner, JD, Vice filled with gratitude for the that fewer than one in 10 grant applications receives Chairman William Michael, CPA, remarkable year we have funding. The Muscular Dystrophy Association, as it Treasurer just had. goes through its process of re-evaluating priorities and Beth E. Johnston, MBA, Secretary In the past 12 months, directions within its strategic planning process, has as E. Ann Biggs-Williams we have launched a series of mid-December put a one-year hiatus on accepting Carol S. Birnbaum, MD Robert H. Brown Jr., MD, of patient support group any new grant applications in basic and translational DPhil* meetings in Baltimore, research areas. This means that organizations like the James A. Chin Sr. June Kinoshita David J. Glass, MD which is livestreamed over FSH Society are an essential lifeline to keep scientists William S. Herzberg the Internet to viewers everywhere. We have sent out working in the FSHD field during these austere and Louis M. Kunkel, PhD Ida Laurello our first ever television public service announcements uncertain times. William R. Lewis III, MD to nine metropolitan markets across the U.S. Over None of these achievements would be possible Michelle H. Mackay, MA Theodore L. Munsat, MD* the summer, the Society recruited FSHD patients to without our FSHD community, the most remarkable Paul Schultz, MD* appear before a governmental committee reviewing and inspiring group I have ever had the privilege to Judith Seslowe, MA Robert F. Smith, Esq. federal policies and funding for muscular dystrophy. meet. I am profoundly thankful to them all: the Soci- Christopher Stenmon, CPA In this issue of FSH Watch, you can read about ety’s staff, Board of Directors, and Scientific Advisory S cientific Advisory Board some additional fruits of our labors. We have assembled Board, and the amazing patients and families who David E. Housman, PhD, Chairman our Honorary Board of celebrities who will help raise volunteer their time to help advocate for FSHD, Michael R. Altherr, PhD awareness of FSHD with the general public. On the organize fundraisers, write articles and letters, edit Robert H. Brown Jr., MD, DPhil science front, FSH Society funding contributed to videos, Tweet and Facebook our links … the list is Rune R. Frants, PhD the landmark discovery that the SMCHD1 gene is a endless, and so is our appreciation. David J. Glass, MD Louis M. Kunkel, PhD modifier of disease severity. At our annual international William R. Lewis Sr., MD scientific conference, the feeling of excitement about the Thank you, and Happy New Year! William R. Lewis III, MD Katherine D. Mathews, MD progress in FSHD research was palpable. Theodore L. Munsat, MD* We are upping the ante on our support for research. George W. A. M. Padberg, MD We reached out, and scientists responded with an Paul Schultz, MD* unprecedented number of exciting proposals. To keep Kathryn Wagner, MD, PhD pace, the Society has also raised more money than ever * Board Member Emeritus before, thanks to our dedicated volunteer event organiz- June Kinoshita ers, generous benefactors, and grassroots members. FSH Society Executive Director FSH Watch WIN TER 2014 Morgan Downey, J.D. It is our editorial policy to report on developments regarding FSHD, but we do not endorse any of the drugs, procedures, treatments, Washington DC Counsel therapies, or products discussed. We urge you to consult with your own physician about any medical interventions. June Kinoshita Executive Director [email protected] FSH Watch is published by the FSH Society and distributed by USPS mail and email to its members and supporters. All material is D aniel Paul Perez President, CEO, and copyrighted and may not be reproduced without written permission. To be placed on the mailing list, provide feedback, or propose an Research Programs article for future issues of FSH Watch, please write to: [email protected] Doris Walsh June Kinoshita, Executive Director Office Manager FSH Society [email protected] 450 Bedford Street Patient Resource Call or email the Society Lexington, MA 02420 USA office to contact a member [email protected] of our Peer-to-Peer Team. FH S Society, Inc. Look for us on the internet at: www.fshsociety.org 450 Bedford Street Editors: Daniel Paul Perez and June Kinoshita Lexington, MA 02420 USA 781-301-6060, -6650 Copy Editor: Elaine Alibrandi 781-862-1116 Fax Graphic Design: Leslie Anne Feagley, Boston, Massachusetts www.fshsociety.org Printing and Mailing: Brian Luoma, ShelDon, Duluth, Minnesota 2 • FSH Watch • WINTER 2014 RESEARCH 2013 FSH Society Pioneer Award Honoring George W. Padberg, MD PhD by DANIEL PAUL PEREZ FSH Society, Lexington, Massachusetts he FSH Society is pleased to present FSHD investigation. With his comprehen- Tthe first Pioneer Award for outstanding sive insights on the complicated nature of contributions to research on FSHD to the disease, he helped many researchers and George W. Padberg, MD, PhD. We are clinicians find a way to approach its genet- profoundly grateful for his dedication to ics and clinical aspects, thereby opening it unlocking the mystery of FSHD and for his for further study and development. guidance, concern, and care for patients In addition to pioneering ways to solve and their families worldwide. FSHD, Padberg is also one of the finest The Pioneer Award was formally be- clinician researchers in neurology, skilled stowed at Padberg’s retirement lecture at in his profession and craft, a man of vision, Grote of St. Stevenskerk in Nijmegen, the a consummate networker, and a significant Netherlands. There he has served as head force in bringing disparate groups together of the Department of Neurology at St. to change the status quo. Radboud University Medical Center. Padberg played a deeply significant Before George Padberg wrote his 1982 role as a long-standing member of the FSH doctoral thesis on facioscapulohumeral dis- Society’s Scientific Advisory Board, helping ease, FSHD research was largely unattended to pave the way for the Society to work with to. Padberg’s keen interest in the disease was the international community and advocate due to first-hand experience with a patient. for patient rights, research funding, and George W. Padberg, MD PhD His thesis remains one of the most thorough education worldwide. and best references on FSHD. For this, he truly deserves to be families, clinicians, and researchers owe Padberg established a gold standard in recognized as a Pioneer. All FSHD patients, him the deepest gratitude. FSH Watch NEWS AND EVENTS TEEING OFF for A CURE On September 23, 2013, we hosted our first annual Swing for the Cure golf tournament in honor of Jeff Johnston and in support of the FSHD Canada Foundation.