Families and Experts Gather at 2011 BMD Conference

Participants share stories, learn about new research and get practical advice at third annual Becker MD conference

Brian Tseng, co-director of the MDA clinic at Massachusetts General Hospital in Boston, spoke about clinical care during the 2011 Becker Muscular Dystrophy conference. (Photos by Thomas Neerken)
Article Highlights:
  • Several hundred people affected by Becker muscular dystrophy met with clinicians, researchers and physical therapists at Cedars-Sinai Medical Center in Los Angeles on Aug. 13, 2011, for the third annual Becker muscular dystrophy conference. The conference was webcast and will be archived online.
  • The gathering, sponsored by MDA, Cedars-Sinai Medical Center and BeckerMD.org, included presentations on BMD clinical care, physical therapy, current drug trials and other topics, as well as extensive question-and-answer sessions and informal opportunities for conferees to interact.
by Miriam Davidson on August 18, 2011 - 3:50pm

Community and empowerment were the themes of the third annual Becker Muscular Dystrophy (BMD) conference held at Cedars-Sinai Medical Center in Los Angeles on August 13, 2011.

Some 150 in-person attendees, and at least that many online attenders, engaged physicians, researchers, physical therapists and other BMD experts on a variety of topics, including the latest advances in research, medical management, physical therapy and clinical trials.

The gathering was sponsored by MDA, Cedars-Sinai Medical Center and the website BeckerMD.org.

The webcast of the conference is available for viewing at http://blip.tv/beckermd. (Editor's note: Some videos were recorded at a lower volume, and will require adjusting your computer's volume settings to be heard. If you should experience additional technical issues, please report them to feedback@blip.tv.)

A 'family reunion'

BMD 2011 conference
Speakers updated attendees on current BMD research and studies.

With several interactive panels and informal get-togethers during lunch and breaks, the conference at times had the atmosphere of a family reunion.

Attendees who had met at previous BMD conferences gathered in little groups to share hugs and stories, while others took the opportunity to discuss their or their child’s personal situations with the assembled experts.

Noting that people came from all over the country to attend the event, keynote speaker Christopher Rosa said that for the first time, “there is talk of a national identity — that we are part of ‘BMD Nation.’”

Rosa, who is affected by BMD and uses a power chair, is assistant dean for student affairs at City University of New York, and a member of the MDA Board of Directors.

Rosa said that previous BMD conferences had been “moving, humbling and life-changing experiences” for him and that get-togethers such as these were essential for people with BMD to “be part of something larger than ourselves.”

BMD clinical care advice

Brian Tseng, co-director of the MDA Clinic at Massachusetts General Hospital in Boston, offered some practical, real-life advice during his presentation on BMD clinical care. Among Tseng’s tips:

  • Know your health record — history, medications, doses, most recent ejection fraction (the percent of blood the heart pumps out with each beat).
  • Request and keep personal copies of all medical records. Make backup copies of everything. Put records in a binder and bring them to medical appointments.
  • Know your dystrophin mutation, and get on a dystrophinopathy registry.
  • Find a dedicated primary care physician (PCP). He or she doesn’t have to be a BMD expert — that’s what specialists are for — but must be open to learning and not in a hurry. A PCP should act as the hub of the wheel for the entire clinical care team.
  • Know your insurance coverage. If you have complicated medical needs, get a case manager and be really nice to that person.
  • Advocate for your care. Be persistent and know how to navigate the system. If you're denied coverage, don’t give up. “You must be in battle station mode — but in a nice way — to get the care you deserve,” Tseng said.

Caring for the heart in BMD

One of the most important aspects of BMD clinical care concerns prevention of cardiomyopathy, or heart disease. Beth McNally, one of the world’s leading experts on the subject, said cardiac problems in BMD can vary from mild to life-threatening, and she advised conference participants to have their hearts closely monitored.

Chris Rosa and Bradley Stephenson

Presenter Chris Rosa with attorney Bradley Stephenson of Texas.

McNally, an MDA grantee who directs the Institute for Cardiovascular Research at the University of Chicago, presented the results of several studies showing that common heart drugs like ACE inhibitors and beta blockers are beneficial in people with various forms of muscular dystrophy, including Becker.

“Prevention is key,” McNally said, adding that because these drugs are generally safe and well-tolerated, patients should start taking them at the earliest sign of heart weakness or malfunction.

Some with BMD have electrical conduction difficulties in their hearts, noted McNally, and can benefit from implanted pacemakers.

Exercise: Consult a physical therapist

Jennifer Hernandez, a physical therapist who specializes in muscular dystrophy, gave a detailed presentation on the benefits and risks of stretching and exercising in BMD. She suggested that mild, daily stretching, including massaging and splinting, can help relax tight muscles, but stretching to the point of pain should be avoided.

Hernandez also said aerobic exercise, like swimming and cycling, can be beneficial, but she cautioned people not to overdo it. She also advised extra care when doing strength training, because muscles can tear when, for example, a person lowers a heavy weight.

Audience members were eager to ask Hernandez about what stretches and exercises they or their family members should be doing, but she stressed that because BMD has such a variable course, what is appropriate in one person’s case would be totally inappropriate in another’s.

“Work with a physical therapist, not a personal trainer,” she said. “Use lighter weights and more repetitions, make sure you’re well-hydrated, and don’t wear yourself out. You shouldn’t try to look buff — just fit.”

Research and clinical trials in Becker MD

At the conference’s afternoon session, attention turned to the current state of BMD research and clinical trials. Presenters described recent progress in a form of gene therapy known as exon skipping, which is mostly aimed at Duchenne muscular dystrophy but also may have implications for Becker. Attendees were urged to learn their genetic mutation so they would be able to participate in future trials of gene therapy and other treatment strategies.

Ronald Victor with a conference attendee
Young conference participant with Ronald Victor

Another promising avenue of treatment involves using drugs like Viagra and Cialis to increase blood flow to weakened skeletal muscles.

Ronald Victor, an MDA grantee and physician at Cedars-Sinai Medical Center who co-hosted the conference, explained why these drugs are potentially beneficial for people with BMD, and urged conference attendees to participate in a clinical trial of tadalafil (brand name Cialis) that is currently under way at Cedars-Sinai.

“Cialis will not cure Becker, but it may permit more exercise with less injury and may slow disease progression,” Victor said, adding that one great benefit of the drug is that it already has received FDA approval for treatment of erectile dysfunction and pulmonary hypertension, so getting it approved for another purpose would be much easier than getting approval for a brand-new drug.

As Victor and Tseng pointed out, the expense and time it takes to bring new drugs to market is a considerable barrier to the development of treatments for rare conditions like BMD.  

For that reason, the physicians said, people need to sign up for disease registries, participate in clinical trials and become advocates for themselves and their community.

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