It is
estimated
that 10% of the cases of cystic fibrosis (CF) and 15% of the cases
of Duchenne muscular dystrophy
(DMD) are due to nonsense mutations. PTC has
catalogued over 1,800 distinct genetic disorders where
nonsense mutations are
the cause of the disease in an appreciable percentage of patients. The FDA
has granted PTC124 fast track
designation for the treatment of CF and orphan drug designations for
the
treatment of CF and DMD due to nonsense mutations.
Memorials to people who had Cystic Fibrosis
.
cline ekwall
Having the first CF test and second
multiplexed genetic test behind Roche's (RHHBF.com/cgi-bin/prnh
/20050207/DCM056 )
"As the father of a young woman with CF, I know only too well the
importance
of corporate philanthropy and how a business such as ours can make
a difference in the lives of children
and young adults with this disease,"
said Motenko.
Nonsense mutations are single-point
alterations in the genetic code that
prematurely halt the translation process, producing a shortened
,
non-functional protein.heard rothenberg
SOUTH PLAINFIELD, N."
About PTC Therapeutics, Inc.
sweat baird
Tm Bioscience
receives FDA clearance for Cystic Fibrosis DNA test
About The Cystic Fibrosis Foundation
The mission of the Cystic Fibrosis Foundation is to assure the development
of the means to
cure and control cystic fibrosis (CF) and to improve the
quality of life for those with the disease
.org. "The funding by the MDA is fundamental to the development of PTC124. By applying this approach
,
PTC has advanced its drug discovery programs rapidly from targets to
preclinical and clinical
drug candidates, building a robust pipeline across
genetic disorders, oncology, and infectious diseases
.7 million for the development of PTC124
for the treatment of CF due to a nonsense mutation in the
cystic fibrosis
transmembrane regulator (CFTR) gene.
About Duchenne muscular dystrophy
(DMD)
Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that
causes the loss
of both muscle function and independence.merz eulogy
Food and Drug Administration (FDA) as an in vitro device
(IVD) for diagnostic
use in the United States. Overall, study results showed that all
genotyping
calls that can be made by the Tag-It(TM) CF Kit were made
correctly and reproducibly under the
evaluated conditions with a
precision of greater than 99. More
information regarding Duchenne
muscular dystrophy is available through the
Muscular Dystrophy Association (http://www.cline fibrosis
Tm Bioscience
is located
in Toronto, Ontario. The award will
be utilized to fund the development of PTC124
for the treatment of Duchenne
muscular dystrophy (DMD) due to a nonsense mutation in the dystrophin
gene.
(Logo: http://www.
PTC is a biopharmaceutical company focused on the discovery,
development,
and commercialization of small-molecule drugs targeting post-transcriptional
control
mechanisms. (CFFT) has awarded
PTC Therapeutics, Inc.transplant cystic
"
"Clearance by the FDA of a genetic
assay for cystic fibrosis provides a
highly standardized product for laboratories that offers tremendous
benefits
to the genetic testing industry, physicians and to patients. Additional information about
Tm Bioscience can be found
at http://www. This gene causes the body to produce
abnormally thick
, sticky mucus that leads to chronic and life-threatening
lung-infections and impairs digestion.cff
. a grant of $1.cute remembered
"This clearance establishes Tm as a unique supplier of CF tests to our
rapidly
expanding customer base against such competitors as ABI (NYSE:ABI) and
Third Wave (NASDAQ:TWTI),
" said Greg Hines, President and CEO of Tm
Bioscience. Combined, the
Universal Array and Luminex
instrument enable the rapid production of
flexible, high-throughput, low-cost DNA-based tests. In
addition, the proceeds from the fund-raising at the grand
openings of every new BJ's location also
are donated.PTC Therapeutics Receives $1.5 Million Grant from Muscular Dystrophy Association
newscom
. Peltz, Ph.
About PTC124
PTC124 represents a first-in-class, orally delivered investigational
new
drug for the treatment of genetic disorders due to nonsense mutations. Each year, approximately
20,000
children worldwide are born with DMD (one of every 3,500 male children).baird transplant
The performance characteristics
of
these products have not been established.
CF is a genetic disease affecting tens
of thousands of Americans. PTC124 allows the cellular machinery to bypass the
nonsense mutation
and continue the translation process, restoring the
production of full-length, functional proteins
., President and CEO
of PTC.
About the Muscular Dystrophy Association (MDA)
MDA
is a voluntary health agency dedicated to helping the more than 1
million Americans affected by neuromuscular
disorders through programs of
worldwide research, comprehensive services, and far-reaching professional
and
public health education.heard obituary
9% in a multi-site
study by testing DNA samples obtained from
the Coriell Institute for
Medical Research (Camden, NJ) repository using three different lots
and
three operators. The Company has
commercialized Analyte Specific Reagents(x) and a series
of Tag-It(TM)
tests(xx) for a variety of genetic disorders., PTC Therapeutics, Inc. PTC's compounds
alter these processes by selectively
modulating how RNA is used to produce proteins. In
addition
to CF and DMD, other potential indications under consideration for
PTC124 include hemophilia, neurofibromatosis
, retinitis pigmentosa,
epidermolysis bullosa, and lysosomal storage disorders. In addition to the
MDA grant, Cystic Fibrosis Foundation Therapeutics, Inc.remembered lifelong
S.9% to 100%)., BJ's Restaurants
culminated
its successful Thanks a Million campaign on behalf of the Cystic
Fibrosis Foundation on Saturday
at the All Star Music Bash hosted by Richard
Marx. "It is my
hope that with their continued support
and dedication and that of all our
volunteers and donors, CF will one day soon stand for 'cure found
.D. For more information please visit
http://www.remembered rothenberg
Based on comparison of
2,924 genotyping
calls for the 43 mutations and variants tested for by
the Tag-It(TM) CF Kit, concordance between
the Tag-It(TM) CF Kit and the
reference tests was 100% (95% CI(equal sign)99. All genetic tests
from Tm
Bioscience are based on the Tag-It(TM) Universal Array platform, which
utilizes a proprietary
universal tag system that allows for easy optimization,
product development and expansion. BJ's,
a long-time supporter of the Cystic Fibrosis Foundation, launched
its Thanks a Million campaign in
2004 with the goal of raising $1 million for
cystic fibrosis (CF) research and care. PTC124 represents
a unique opportunity to use a
single small-molecule drug to address chronic and life-threatening
diseases of
high unmet medical need.eulogy transplant
This DNA based test is used to simultaneously detect
and identify
mutations and variants in the cystic fibrosis transmembrane conductance
regulator
(CFTR) gene in human blood specimens in order to determine CF
carrier status in adults, as an aid
in newborn screening, and in confirmatory
diagnostic testing in newborns and children.
It is estimated
that 15% of the cases of DMD are due to a nonsense mutation. We commend PTC for their use of innovative
treatment approaches for genetic diseases like Duchenne muscular dystrophy,"
commented MDA President
and CEO Robert Ross. Phase 2 studies in patients with Duchenne muscular
dystrophy (DMD) and cystic
fibrosis (CF) are planned for 2005.org).memorials eulogy
Cystic Fibrosis (CF) is the most common autosomal recessive
disorder in
the Caucasian population, with an incidence of approximately 1 in 3,200 live
births
. Single-dose and multiple-dose
Phase 1 studies involving healthy volunteers have been recently completed
and
PTC is working with investigators and the FDA to finalize plans for Phase 2
studies in DMD
and cystic fibrosis.ekwall cline
Performance testing has
established that the Tag-It(TM) CF Kit operates with
100% accuracy and greater
than 99. "The Tag-It(TM) Cystic Fibrosis Kit is the only CF testing system
that has performance characteristics which have been established through
extensive studies reviewed
by the FDA. I hope that we
can continue to count on their support until this disease is defeated
. They are a truly outstanding partner in our mission to
defeat this life-threatening disease," said
Gary Greene, executive director of
the Southern California Chapter of the Cystic Fibrosis Foundation
.
"We are honored to receive this grant from the MDA and appreciate their
continued support
of PTC124," said Stuart W. PTC124 allows the cellular machinery to bypass the
nonsense mutation
and continue the translation process, restoring the
production of full-length, functional proteins
.cute angie
9%.
"On behalf of the CF Foundation, I would like to express our gratitude to
BJ's Restaurants
.transplant merz
Analytical and performance characteristics
are not established.newscom.
(PTC), a biopharmaceutical
company focused on the discovery, development, and
commercialization of small-molecule drugs targeting
post-transcriptional
control mechanisms, today announced that the company has been awarded a $1.5
million grant from the Muscular Dystrophy Association (MDA).
transplant baird
Michael
Watson, Executive
Director, American College of Medical Genetics.
About Tag-It(TM) reagents and genetic tests
Tm Bioscience's product menu is focused in the fields of human genetic
disorders, pharmacogenetics
and infectious disease.
(x) Analyte Specific Reagent.
(Photo: http://www. "I am
proud of BJ's dedicated support of the CF Foundation and
am grateful to all those who contributed
to the success of the Thanks a
Million campaign, especially our employees and our customers.
Currently
, there are more than two-dozen potential therapies for CF in the CF
Foundation's drug development
pipeline, any one of which could have a profound
effect on the length and quality of life of those
with CF. For more information on CF and
the CF Foundation, call (800) FIGHT CF or visit http://www
.
Nonsense mutations are single-point alterations in the genetic code that
prematurely halt the
translation process, producing a shortened,
non-functional protein.
"This award through MDA
's Translational Research Program represents a
major commitment by the Association to partner with
industry in the
development of new drugs.mortality cline
- FDA grants first IVD clearance to Tm Tag-It(TM) CF
multiplexed genetic
test -
TSX: TMC
TORONTO, May 9 - Tm Bioscience Corporation
(Toronto, Ontario; TSX: TMC), a leader in the commercial genetic testing
market, is pleased to
announce that its Tag-It(TM) Cystic Fibrosis (CF) Kit is
the first multiplexed human disease genotyping
test to be cleared by the U.BJ's Restaurants Gives Million Dollar Thank You to Cystic Fibrosis Foundation
at All Star Music Bash Hosted by Singer/Songwriter Richard Marx
"
BJ's Restaurants is an ardent
supporter of the CF Foundation's CF research
and care programs through Cookies For Kids, a cause
related marketing campaign
in which it donates a portion of its Pizookie dessert sales to the CF
Foundation.obituary memorials
PK) AmpliChip CYP450 to be
cleared as an IVD, sets the regulatory pathway for other
tests in our broad
and growing pipeline and positions Tm as a leader in the commercial genetic
testing
market." said Dr.
Precision was tested using synthetic positive controls representing all
potential genotyping calls for all 43 mutations and variants tested for
by the Tag-It
(TM) CF Kit.com.
HUNTINGTON BEACH, Calif. The $1 million total
donation was generated
from these programs as well as donations from BJ's
employees, vendors and guests. DMD is perhaps
the
most prevalent of the muscular dystrophies and is the most common lethal
disorder diagnosed
during childhood today.ekwall cystic
The Tag-It(TM) Cystic Fibrosis Kit simultaneously screens for the
23 cystic
fibrosis transmembrane conductance regulator (CFTR) gene mutations
and 4 variants (polymorphisms
), as recommended by the American College of
Medical Genetics (ACMG) and the American College of Obstetricians
and
Gynecologists (ACOG) in 2004.
(xx) For Investigational Use Only. BJ's co-Chairman of the
Board Paul
Motenko presented the $1 million check to Richard Marx, who accepted it on
behalf of
the CF Foundation.J. We feel privileged to work with
the MDA, and share their commitment to finding
a treatment for DMD.mdausa.org.npr cf
Tm Bioscience's product pipeline includes tests for genetic
disorders
, drug metabolism, and infectious diseases.'"
The CF Foundation strongly believes that an investment
in CF medical
research is an investment in the future of all people with the disease.mdausa.rothenberg mortality
9% reproducibility
and precision. In addition, the kit screens for 16 additional
mutations prevalent in North America
or the world.
The kit is not indicated for fetal diagnostic, pre-implantation testing
or
for stand-alone diagnostic purposes.tmbioscience.cute heard
Performance Results
The Tag
-It(TM) Cystic Fibrosis Kit is sold with the following FDA cleared
performance characteristics:
Accuracy of the Tag-It(TM) CF Kit was established in a method comparison
study using DNA
sequencing as reference methods.
Reproducibility was established as greater than 99. Assays
from Tm operate on the Luminex
xMAP(R) system, a well-established bead based instrument. More
than
ten million Americans, or approximately one in 31 people, are unknowing
carriers of the defective
CF gene.
The MDA is among the most active associations in the United States supporting
research
from scientists across multiple disciplines in an effort to identify
new treatments for neuromuscular
diseases. Post-transcriptional control processes are the sequence
of events in the cell that ultimately
regulate the rate and timing of all
protein production.baird remembered
About Tm Bioscience - Putting
the Human Genome to Work(TM)
Tm Bioscience is a DNA-based diagnostics company developing a suite
of
genetic tests.com/cgi-bin/prnh/20010919/PTCLOGO )
PTC124 represents a first-in-class, orally
delivered investigational new
drug for the treatment of genetic disorders due to nonsense mutations
. PTC124 has demonstrated the
ability to restore full-length functional protein in genetic disease
models
harboring nonsense mutations.transplant richman
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