13
Exon Skipping as a Class
Dear FDA, Our son was diagnosed just 5 years ago
at age 9. We consider our family to be lucky because
in the past 5 years Duchenne has only taken away
our son’s walking, standing, dressing and bathroom
independence. So far we have managed these things.
We are also lucky because in the past 5 years we’ve
only had to purchase (out of pocket), one manual
chair for travel, one power chair, one ceiling mounted
lift, one freestanding lift for travel, one mini-van with
turning seat and lift, one mini-van with manual ramp
and lockdown element, one-one story house with
wide doorways and halls, a couple of doorway ramps
and one suitcase ramp for travel. Not sure how, but
we have managed these things as well. We are also
lucky to be ghting Duchenne in the past 5 years
since there are many excellent scientists working
internationally to nd ways to treat everyone with
Duchenne. We are doing our part to make things
work for all our sons. Now it’s your turn. Approve
Exon Skipping as a “class” instead of making each
and every exon skipping compound go through
years and years of individual clinical trials. Approving
exon skipping as a “class” may not be risk free but
under the circumstances it is still the right thing to
do. Do the right thing so we can have a realistic
hope that our son(s) will live long enough to see
regenerative medicine. Do the right thing so they will
have the chance to stand, walk, dress or bathroom
independently. Do the right thing because if you were
in my shoes, you’d want the same thing for your own
son. They deserve nothing less. Thank You.
Need to Look at Duchenne Differently
than Other Diseases
I want the FDA to know that as a mother with a child
with Duchenne, we are desperate for any treatment
that can help our son to be stronger or live longer.
We have a different requirement for safety and trials
of the medications/treatments. This is not a regular
disease. Drugs should always be fast tracked. There
needs to be a different set of requirements. This is
imperative for our family’s well-being.
Three Sons with Duchenne in One Family
My name is Betty Vertin. I live in Hastings, NE with
my husband and ve children. We have a 12 year old
daughter Lexi and 4 sons; they are Max, 8, Chance,
6, Rowen, 5, and Charlie, 3. In July of 2010, when I
was just 10 weeks pregnant with our youngest, our
son Max was diagnosed with Duchenne. In a matter
of minutes a specialist told us what no parents ever
want to hear: that our son would be wheelchair bound
by 9, need our help with activities of daily living as
a teenager, and would die before his 20th birthday.
We woke up that morning with a beautiful healthy
son and went to bed that night grieving the loss of a
son that would come too soon. In the course of the
next year, Rowen and Charlie were given the same
fatal diagnosis. We believe, or at least hope the
progression of the disease may not happen as quickly
as the rst doctor described to us; but we do know
there is no cure and our sons will die because of the
disease if there is no cure or at least better treatment
options. I am sharing with you because three of our
four sons have Duchenne Muscular Dystrophy. I am
sharing with you because I am scared that 3 of my 5
children could die without a cure or better treatments
of Duchenne Muscular Dystrophy. My sons are young;
but still face daily challenges because of Duchenne
Muscular Dystrophy. Max is our oldest son, he is
8. Max’s muscle strength is in a decline. There are
things he could do three or four months ago that
are becoming increasing more difcult for him. He
is falling more often. Two weeks ago he fell and hurt
the tendons around his knew. He limps and uses his
medical stroller more now than ever before. We hope
that as his tendons heal, his mobility will come back.
However, because of Duchenne Muscular Dystrophy
there is no guarantee that he will bounce back. Just
yesterday he fell trying to play soccer, hit is head and
has a concussion. He was falling like this three months
ago. The hardest for me to swallow is that Max does
not always nish putting a lego set together. That was
unheard of a couple of months ago; Max would sit for
hours to complete a set, but now he doesn’t seem
to have the stamina. As a parent this is very hard to
watch, no one should watch their children regress.
Parents dream of watching their sons grow into men,
watching their dreams come true. Duchenne and the
lack of treatment threatens to steal this dream from
us. It threatens not just the loss of one son, but three
sons. It threatens to take half of my children from me.
My husband and I feel we are proactive, we are doing
everything within our power to save our children. We
make nancial sacrice to take our children to the
best doctor available (14 hours from our home), we
follow a strict diet, we home stretch, we fund-raise
to help nance research. In fact, Max participates in
the [clinical research] study and [drug] trial in hopes