Courage and Persistence

The two most powerful weapons are courage and persistence. Nothing can stop a person armed with both. - Scott Sorrel

Armed with courage and persistence the advocates of the Dystonia Advocacy Coalition (DAC) have worked tirelessly these past few years and finally achieved our goal of having dystonia included on the list to be eligible for funding through the Congressionally Directed Medical Research Program. This means, because of the efforts of our dystonia advocates, our researchers will have another source of potential funding – getting more dystonia research going! Dystonia advocates have walked the halls of Congress – meeting with Members and their key staff; met multiple times with Congressional Staff in local offices; and have written letters and made countless calls to make this happen – AND THEY DID IT!

The DMRF has proudly worked with advocates from the Benign Essential Blepharospasm Research Foundation, DySTonia, Inc., the National Spasmodic Dysphonia Association and the National Spasmodic Torticollis Association for over four years to address the legislative and policy needs of the dystonia community. Congratulations to everyone who had a part in this. You have demonstrated that our working together – collaborating on this critical effort – makes a difference and we all have something to celebrate.

Please plan on joining us as we continue our fight for dystonia. The DAC Advocacy Day is scheduled to take place in Washington, DC on May 5 with a training and orientation session planned for May 4th. We look forward to having you there.

Register for Advocacy Day by visiting the following link:
http://www.rsvpbook.com/event.php?470398

Myoclonus Dystonia Meeting

Pictured above from left to right: Thomas Gasser, MD, Marie Vidailhet, MD and Susan Bressman, MD

This past weekend the DMRF hosted a meeting on myoclonus dystonia, a form of dystonia with symptoms that include rapid jerky movements, alone or in combination with the sustained muscular contractions and postures. We were fortunate to have the leadership of Drs. Susan Bressman and Marie Vidailhet in developing the agenda and bringing together the best faculty in the world to discuss the status of myoclonus dystonia research, clinical and basic, and to develop a plan for keeping the momentum of progress moving forward. Joining the DMRF for this incredible meeting was esteemed researchers from France, Germany, Italy, the Netherlands, the United Kingdom, Canada and the USA. The agenda included a review of genetics, imaging and neurophysiology, therapeutics, and animal models with a discussion on future directions.

In summarizing the meeting, Dr. Bressman said this was the best workshop she had been involved in for many years. We felt the same way – the energy, the enthusiasm and the spirit of collaboration was amazing and we look forward to implementing the plan for attacking myclonus dystonia.

Having these kinds of meetings is an important part of the DMRF’s scientific strategy – implemented and operated by our full time Science Officer, Jan Teller, PhD, and our Medical and Scientific Director, Mahlon DeLong, MD of Emory University. Supporting research, awarding fellowships and contracting for specific scientific work is very important but so too are these meetings when we are able to bring great minds together to discuss a specific topic and develop next steps.

We are so thankful to all who participated in the meeting and to everyone in the dystonia research community for their dedication and commitment to ending all forms of dystonia.

The DMRF: A Critical Piece of the Dystonia Puzzle

Starting a scientific career is a daunting proposition, especially for a physician-scientist that has clinical as well as lab responsibilities. Yet, it is physicians who witness first hand the struggles of patients, and this knowledge can be a powerful motivator for scientific discovery -- even when the underlying science is unknown and therefore “off the radar” of basic investigators.

The DMRF has provided essential support to my scientific career, both at the critical early stage, as well as at later stages. Shortly after the gene encoding torsinA was discovered, I received a grant from the DMRF to generate torsinA mutant animals. This grant - to a young and untested physician-scientist – was helpful in supporting the work but, perhaps as importantly, gave me an early track record of securing funding and was therefore helpful in me getting other grants, both from the Howard Hughes Medical Institute as well as the NIH. The ongoing support of the DMRF also enabled me to hire and support Rose Goodchild, an outstanding young scientist who now has an independent lab of her own (studying torsinA). Moreover, the DMRF has consistently organized and supported basic science and clinical workshops that have been invaluable in helping to integrate and focus the dystonia research community. I have found these meetings invaluable, both scientifically and as a place where trainees in my laboratory (and others) can begin to present their work and develop confidence as scientists. The DMRF has consistently focused on identifying and supporting the best science, and I’m convinced that we’d be far, far further from our goal of improving the treatment and hopefully cure of dystonia without the many valuable DMRF-supported activities.

Guest Blogger:

William Dauer M.D.
Elinor Levine Associate Professor
Neurology & Cell and Developmental Biology University of Michigan Medical School

Would you like to know more about DMRF grant funding opportunities?
http://www.dystonia-foundation.org/pages/funding_opportunities/142.php

The deadline for all applications is December 15, 2009. Visit http://www.dystoniagrants.org to view the application form.

Dystonia Researchers

Pictured above are Rose Goodchild, PhD, University of Tennessee and Cris Bragg, PhD, Massachusetts General Hospital

Attracting and keeping smart, dedicated young researchers is really important. Recently the DMRF hosted a meeting of young investigators, bringing together some of the brightest young dystonia researchers to talk about dystonia research and how best to support their work. We wanted to hear from them as to the challenges and opportunities they are experiencing – and how they thought the Foundation could best help young dystonia scientists. We also wanted their ideas on new, perhaps innovative ideas for DMRF research support. It was a very productive meeting with a great exchange of ideas for how we can move forward into 2010 and beyond.



This week the Society for Neuroscience is meeting in Chicago. This is an amazing meeting and in the past few years – dystonia has been present in poster sessions and talks. Having a dystonia presence at this meeting is an accomplishment for all to celebrate. DMRF, along with NSDA, had a booth at the meeting – giving us the opportunity to further promote dystonia at this meeting.



Last evening the DMRF hosted an event where we brought together many members of the dystonia research community – senior level investigators with younger investigators – all talking about what is new in dystonia research, what steps we should look at next and the progress that has been made in the last few years. It was wonderful to see the dialogue between these generations of dystonia researchers, to witness the established investigators encouraging the thoughts and ideas of those investigators beginning their careers.

NIH Awards New Grant to Develop Better Treatments for Focal Dystonias

Pictured above are H. A. Jinnah, MD, PhD and Joel Perlmutter, MD

We are pleased to tell you that the National Institutes of Health (NIH) have announced the funding of a five year award aimed at forming a multicenter Dystonia Coalition to advance clinical research on primary focal dystonias. This includes Cervical Dystonia, Spasmodic Dysphonia, Blepharospasm, and others. Leading the Coalition will be H. A. Jinnah, MD, PhD, of Emory University and his co-director is Joel Perlmutter, MD of Washington University. The Coalition will bring together the most committed dystonia researchers in North America and Europe, along with dystonia patient advocacy groups. The Dystonia Medical Research Foundation (DMRF) is proud to play an integral role by providing logistical and planning support for the Coalition. The first meeting of the Coalition is scheduled for November 5th.


The $6 million award will allow the Dystonia Coalition to cultivate a better understanding of the primary focal dystonias and find better therapies. This includes projects to develop a better understanding of their natural history, establish instruments appropriate for monitoring disease severity in clinical trials, and develop proper diagnostic criteria.

Dr. Perlmutter will lead the effort to create a biorepository to store biological samples to support future research, making these resources available to investigators worldwide. Cindy Comella, MD of Rush University Medical Center, will lead the effort to develop a comprehensive rating scale, including assessing quality of life, for Cervical Dystonia and Christy Ludlow, PhD of James Madison University, will be leading the effort to develop tools to diagnose and measure severity of Spasmodic Dysphonia and assess its impact on quality of life.

In addition to these important efforts, this program will support the development of pilot projects that will directly impact diagnosis and therapy development for focal dystonias. Also included in this effort will be the awarding of career development grants to support young investigators in dystonia research.

This is a huge accomplishment for the dystonia community. This award reflects a great deal of work and collaboration among the dystonia research and patient organizations. The work done through this award is so important to helping prepare the community for clinical trials – when new, potential treatments are available. This is something that should be celebrated by all!

2009 Child Neurology Foundation Advocacy Award Recipient

It is with great pride that I announce that DMRF has been selected as the 2009 Child Neurology Foundation’s Advocacy Award of Merit recipient. We are thrilled to receive this award from CNF, an organization that is dedicated to advocating for children and adolescents with neurologic disorders. In announcing the award, CNF states, “The Dystonia Medical Research Foundation demonstrated innovativeness and achieved phenomenal impact on behalf of the greater community. It gladdens us that we can honor the organization nominated by Dr. Jonathan Mink. DMRF is regarded as one of the strongest organizations providing advocacy while also engaging the scientific community to pursue research.”



To receive this kind of recognition for our work is a great honor. Claire Centrella, DMRF’s President, and I will receive the award next month on behalf of every member of the DMRF community. It is because of our members and supporters that the DMRF is able to fund cutting-edge research and provide support programs for children and adults who are battling dystonia. You all share in this award.

Thank you!

Insurance Survey Update

We want to thank everyone who participated in the insurance survey last month. We very much appreciate the time and thought you put into your responses and we have over 900 people take time to help us identify the insurance issues facing the dystonia community. Thank you all! We will let you what we found and our plan for addressing the issues in the near future.

I want to thank Oxana Zabelina for all of her work in creating and implementing the survey and the issues facing those who are fighting dystonia. Oxana, a fighter herself, has returned to the University of Massachusetts, and has resumed her studies there. We wish her every success and are grateful to her for helping us make this happen.

We also want to thank our sister organizations that worked to refine the survey and promote participation. NSDA, BEBRF, NSTA and DySTonia, Inc. were great and we are proud to work in partnership with them on activities that will help all who suffer from dystonia.

Thank you everyone!