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Parent Project Muscular Dystrophy Awards UCLA End Duchenne Grant

    Dr. Carmen Bertoni to Receive $75,000 For Duchenne Muscular Dystrophy
Research

    MIDDLETOWN, Ohio, Nov. 6 /PRNewswire-USNewswire/ -- Patricia A.
Furlong, Founding President and CEO of Parent Project Muscular Dystrophy
(PPMD), the largest non-profit organization in the United States focused on
finding a cure for Duchenne muscular dystrophy (Duchenne), announced today
the latest recipient of the End Duchenne Grant Award Program. Carmen
Bertoni, Ph.D., Assistant Professor of the Department of Neurology at the
University of California - Los Angeles will receive a grant for $75,000, to
be used to continue her work with high throughput screening (HTS) to
identify chemical compounds that increase the efficiency of
oligonucleotide-mediated (ssODN) gene correction in Duchenne muscular
dystrophy ("Duchenne") and that can be advanced into a clinical application
for the treatment of Duchenne.

    The End Duchenne Grant Award Program was created by PPMD in partnership
with the National Institutes of Health (NIH) in an effort to ensure
continuation of promising Duchenne research and translation to human
studies. The End Duchenne Grant Award Program is a bridge grant provided by
PPMD to selected research projects that receive scores beyond the current
funding paylines of the NIH Institutes and Centers supporting Duchenne
research. The first grants were awarded earlier this year.

    Explains Ms. Furlong, "With its research budget growing tighter each
year, the NIH can fund only a fraction of the many promising applications
it receives. Currently, only those grants which score in the top
percentiles are able to be funded."

    The End Duchenne Grant Award Program represents the first Duchenne
specific bridging program and the first ongoing bridge funding program to
be presented in the rare disease category. As a collaborative effort with
the NIH, the selection strategy behind this award is very different than
any other private research investment in Duchenne, yet the goal of this
award remains consistent with all of PPMD's research initiatives: to
advance promising research which will impact this generation affected by
Duchenne.

    The recipient of an End Duchenne Grant must be focused on translational
research (the process of applying ideas, insights and discoveries generated
through basic scientific inquiry to the treatment or prevention of disease
or injury). The NIH will notify investigators whose translation grant
applications score well, but not within the NIH funding levels, to submit
their applications and score sheets to PPMD. H. Lee Sweeney, PPMD's
Scientific Director and a member of the Grant's Review Board says, "PPMD's
Scientific Review Board will re-review these applications seeking to
identify those with the greatest potential to ultimately help all boys with
Duchenne."

    A researcher at the University of Pennsylvania himself, Dr. Sweeney
went on to say that "Interest in the End Duchenne Grant Program is
continuing to grow tremendously. Scientists have renewed faith in their
projects because an organization like PPMD is showing their support by
providing bridge funding. The quality and promise of the entries we are
reviewing is impressive to say the least. UCLA received funding earlier in
the year for the work that Dr. Melissa Spencer is doing. We are thrilled
that Dr. Bertoni has also chosen Duchenne as the focus of her talents. If
we can continue to help scientists like Dr. Bertoni maintain the momentum
of these important avenues in treatment, so that they can receive funding
from the NIH in the near future, then we will consider this grant program a
huge success."

    Dr. Bertoni's proposal will screen the Prestwick library, a collection
of all of the FDA approved drugs to identify compounds that can
significantly enhance the ssODN-mediated gene repair and that are already
approved for use in humans, thus increasing the chances of moving this
technology into a clinical scenario in a relatively short period of time.

    Says Dr. Bertoni, "It is a tremendous honor to have the faith and
support of the families fighting Duchenne. I believe my work will help
speed up the approval process for getting critical technology to the trial
stage and because of the financial support of PPMD, we can continue moving
our work forward."

    The End Duchenne Grant Award Program was designed to enable
investigators to continue their projects and generate additional data for a
successful re-application within a 12 to 24 month period, thus leveraging
additional Duchenne-specific research dollars.

    Duchenne, the most common form of childhood muscular dystrophy, is a
progressive and fatal muscle disorder affecting boys and young men that
causes the loss of muscle function, wheelchair dependency and a decline in
respiratory and cardiac function.

    An applicant seeking support from the End Duchenne Grant Award Program
must be employed at a for-profit or non-profit organization or institution
and have submitted and received a complete review of an application to the
NIH directed toward translation of research into human clinical studies
specific for Duchenne muscular dystrophy. The applicant must have the
resources to conduct the proposed research project and the
organization/institution must have appropriate grant administrative
capacities for the handling and disbursing of research funds. For more
information or to apply, please visit
http://www.parentprojectmd.org/EndDuchenneGrant.

    About PPMD

    Parent Project Muscular Dystrophy (PPMD) is a national not-for-profit
organization founded in 1994 by parents of children with Duchenne and
Becker muscular dystrophy. The organization's mission is to improve the
treatment, quality of life and long-term outlook for all individuals
affected by Duchenne muscular dystrophy through research, advocacy,
education and compassion. PPMD is headquartered in Middletown, Ohio with
offices in Fort Lee, New Jersey. For more information, visit
http://www.parentprojectmd.org.