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Journal of Research and Reports in Gynecology and Obstetrics | Volume: 3
February 28-March 01, 2019 | Paris, France
Palliative Care, Obstetrics and Gynecology
Stroke and Clinical Trials
International Conference on
Joint Event on
International Conference on
&
C
linical trials in the pediatric neurology patient population
are very common in the last decade. There are clinical trials
reflecting rare forms of epilepsy and neuromuscular diseases.
Few examples are provided below:
Dravet syndrome (DS) or severe myoclonic epilepsy in infant
is one of the most well-known disorders of the epileptic
encephalopathies. DS is a highly treatment-resistant and
refractory epilepsy syndrome. Establishment of a seizure free
condition in affected children, even with anticonvulsant drug
polypharmacy is extremely rare. Lennox-Gastaut Syndrome
(LGS) is a rare epileptic encephalopathy. Onset of LGS usually
occurs before age 11, with a peak between 3 and 5 years of
age. Nearly all LGS patients have treatment-resistant, lifelong
epilepsy, with a poor prognosis. Duchenne muscular dystrophy
(DMD) is a disabling and life-threatening X-linked genetic
disorder affecting males. Boys with DMD develop progressive
proximal muscle weakness that leads to deterioration of
ambulation, wheelchair dependency, and eventual respiratory
and cardiac failure. Spinal muscular atrophy (SMA) is an
autosomal recessive neuromuscular disease resulting in atrophy
of the voluntary muscles of the limbs and trunk. It is the most
common genetic cause of infant mortality, and a major cause of
childhood morbidity in the U.S.
Clinical trials in pediatric neurology rare diseases are testing
new investigational drugs (ids). There is still an ethical dilemma
if the new treatment will be better than the standard of care.
There are number of unknown risks. Patient’s enrollment can
be very challenging. The commitment from the families is huge.
Most of the clinical trials start with the double-blind, placebo-
controlled design. Families have to agree on the possibility for
their child to be randomized in the placebo arm. Those clinical
trials might have very frequent study visits which might cause
financial burden. Lot of work needs to be done to address the
ethical challenges.
Speaker Biography
Vesna Popovska has graduated from medical school in 1988 in Skopje, Macedonia. She
completed her residency in obstetrics & gynaecology in 1998 inMacedonia. She moved
to Canada in 1999 and joined the Division of Maternal Fetal Medicine (MFM) at BC
Women’s Hospital, where she built, established and lead the entire research program
as a program manager. In 2005, she was recruited by the neurosciences program at BC
Children’s Hospital, as a senior research manager. She developed and led the program,
becoming its director in 2016. She is involved in the strategic planning, implementation
and evaluation of the program activities and the management of multiple complex
projects. She is responsible for recruitment, supervision and evaluation of all
research staff at neurosciences program. She continues to lead the program,
develops collaborative relationship among universities, industry partners and Clinical
Research Organizations (CROs) to support and advance patient-oriented research.
e:
vpopovska@cw.bc.caVesna Popovska
BC Children’s Hospital, Canada
Ethical challenges in pediatric neurology clinical trials
Vesna Popovska
, Res Rep Gynaecol Obstet, Volume 3
DOI: 10.4066/2591-7366-C1-001