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DMD Family Roundtable Focused on "Daily Care of the DMD Child"

 Saturday, May 3 - 8:30 a.m - noon Massachusetts General Hospital

    The roundtable format was very interactive.  Families came prepared to ask lots of

    questions and to share their own experiences and wisdom about caring for the whole

    child and the family.

   Presenters were:

  • Brian Tseng, MD, PhD - Director of Jett Program for Pediatric Neuromuscular Disorders at MassGeneral Hospital for Children:   Clinical research update
  • Christine McSherry, RN, President of Jett Foundation:  Total care of the DMD child
  • Tracey Denell, LICSW - Specializes in working with children with serious illnesses and their families:  Navigating the emotional and social challenges of DMD for the entire family
  • Tracy Seckler, Co-founder of Charley's Fund:  New inroads in family-funded research
Christine McSherry
Family Advocate

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Tips on Caring for Your Child with DMD

    Here are some helpful hints.  Please let
    me know if you have questions or
    would like more information on these.
  • 10 hours a sleep per night is optimum.
  • Complaints of chest pain should be taken seriously.  Try to have a recent ekg with you if you take him to the ER or physician's office.
  • Monitor your child's weight, encourage healthy eating habits.
  • Snoring at night/tiredness in the day may need to be addressed with a sleep study.
  • Night casts should be worn even if child's heel cords don't appear tight.  Encourage this good habit early.
  • Boys with a fractured femur may benefit  from surgery rather than casting.
  • Metal tacks placed in the heel of the sole of your son's shoes can serve as an auditory reminder to land on heels first.
  • Depression and OCD behavior seem to be a common thread, not your fault or his. Experienced therapists can offer some solutions.
  • Elevated liver function test on your son's labs do not necessarily indicate liver failure.  Please consult an experienced DMD physician before consenting to a liver biopsy.
  • Alert anesthesia team that your son has a neuromuscular disorder and may be at  risk for malignant hypothermia.
Tracey Denell

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Talking to your kids
  • They (DMD kids) need a story to tell.
  • The story should be one they understand and in their words.  They need to be comfortable with telling their story and not get upset when telling it.
  • The story needs to be in language others will understand.
  • They need to tell others how they are different and how they are the same. 
  • Encourage good relationships among all of your children, which will serve as a model for how kids outside the family treat your child with Duchenne.  Allow your healthy children to be separate from their sibling's diagnosis so they don't resent their brother and you.
  • Do not keep the diagnosis a secret - I have seen this be emotionally tragic in later years.  They are smarter than you think.  You can't keep things from siblings for long and need to be in the loop.  They worry too.
  • Treat your children's relationship as normal as possible.  Let them fight and love.  Don't interfere too much - they need to work out their own relationship.  Don't take sides if you can help it.  They will be more resilient and emotionally healthy for that.  Don't force the relationship and don't force the sympathy it will only make them more resentful.  They will surprise you!!  Ex. I wish I was in a wheelchair - attacking the doctor.  Getting on the boat, fear.
  • Siblings need their own story.  The world should not always revolve around the DMD child.  Find the specialest in each.  Work on your guilt!!  Treat them normally.
Tracy Seckler
Charleys Fund

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  • Charley's Fund works with Transnational Research.
  • From lab to human clinical trials.
  • Raised over 6 million.
  • Have committed 12 million thru 2010
  • We act as a catalyst to ensure medical research moves quickly to save this generation.
  • Funding at 18 Universities.
  • Our Director is the only PhD in the world whose sole job is to expedite finding a treatment.
  • Children's National Medical Center in Washington, DC (we scour the world).
  • Run thru MDX mouse is it or is it not helpful - shown promise - put them to the test.
  • For current research go to Charleysfund.org
Charleysfund.org
Dr. Brian Tseng
Director of JPPND
drbrian

  
    General observations regarding
    Estimates of Funding Research:
  • $70 million in research for DMD - compared to $5,654 million for cancer
  • US tax dollars are paying for this
  • We need a major shift to change this
    Four stages of research prior to a new
    drug on the market from Dr. Brian:
 
    Phase I   Designed to establish the
                   effects of a new drug in
                   humans.  These studies are
                   usually conducted on
                   small populations of healthy
                   humans to specifically
                   determine a drug's toxicity,
                   absorption, distribution and
                   metabolism.

    Phase II  After successful completion of
                   Phase I trials, a drug is then
                   tested for safety and efficacy
                   in a slightly larger population
                   of individuals who are
                   afflicted with the disease or
                   condition for which the drug
                   was developed.

    Phase III The last pre-approval round of
                   testing of a drug is conducted
                   on large populations of
                   afflicted patients.  Phase III
                   studies usually test the new
                   drug in comparison with the
                   standard therapy currently
                   being used for the disease in
                   question.  The results of these
                   trials usually provide the
                   information that is included in
                   the package insert and
                   labelling.

   Phase IV After the drug has been
                  approved by the FDA, phase IV
                  studies are conducted to
                  compare the drug to a
                  competitor, explore additional
                  patient populations, or to
                  further study any adverse
                  events.

    Dystrophin - spelling of biggest gene

    "DMD can present genetically in four
    different ways, knowing your child's
    genetic disposition will help when
    searching out clinical trials for
    treatment"

    1.     Exon deletion
    2.     Exon skipping
    3.     Stop code signals PTC - (tricks
            system to ignore stop) Stopping
            too soon
    4.     Exon duplication

    Four DMD Goals:

    1.     A cure with minimal side effects
    2.     Better treatments with minimal
            side effects
    3.     Dedicated care to optimize
            quality/quantity of life
    4.     Minimize/Reverse scar tissue in
            muscular dystrophy

    While we wait:

    1.     Best supportive care possible
    2.     Avoid damaging things
    3.     Ramp up on favorable things

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Dr. Brian Tseng                       Christine McSherry

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Tracy Seckler                           Christine McSherry &
                                                Tracey Denell
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Kids Group Portrait