Toxic Strand Of RNA Causes Myotonic Muscular dystrophy
Studies have indicated that myotonic
dystrophy in adults, which is the most common type of myotonic
dystrophy could be due to a toxic RNA. // br>
Doctors at the University of Virginia Health System have shown for the first time that by getting rid of poisonous RNA (ribonucleic acid) in muscle ce...
Insmed Provides Update on Supply of IPLEX(TM)
...supply these patients for no more than 24 months.
The Company intends to analyze the on-going data collected for various indications, including myotonic
muscular dystrophy and ALS, and assess the overall IPLEX(TM) development program, including possible IPLEX(TM) manufacturing options with third partie...
Scientists Reverse Muscular Dystrophy in Mice
...that you can impact the molecular underpinnings of myotonic
dystrophy, which is an important step forward in t...lying causes," she said.
According to the MDA, myotonic
(muscle-stiffening) dystrophy is one of nine forms...ases which primarily affect voluntary muscles.
dystrophy type 1 is the most common form of muscul...
Muscular Dystrophy Association Launches New Venture Philanthropy
...uchenne muscular dystrophy (DMD); amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease); spinal muscular atrophy (SMA); and Friedreich's ataxia, myotonic
dystrophy, and limb-girdle muscular dystrophy.
MVP will approach the raising, investment and management of funds much like a venture capital fir...
Scientists exploring new compounds to target muscular dystrophy
...lecule that targets the wayward RNA at the root of myotonic
muscular dystrophy," said Benjamin Miller, Ph.D., ...s shown how a genetic flaw creates the symptoms of myotonic
dystrophy, which affects about 35,000 Americans. ... first step toward developing a drug treatment for myotonic
dystrophy. The message from our patients is loud a...
Insmed Gains Royalty-Free Worldwide Rights for IPLEX(TM) in Connection with Potential Expanded Access ALS Programs
... 2005 for the treatment
of children with growth failure due to severe primary IGF-I deficiency, is
currently being investigated in ALS in Italy and in myotonic
Insmed Inc. is a biopharmaceutical company with unique protein process
development and manufacturing experience a...
JCI table of contents: Nov. 15, 2007
... patients with the most common form of muscular dystrophy affecting adults, myotonic
dystrophy type 1 (DM1), also experience an inability to voluntarily relax t... splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic
Charles A. Thornton
University of Rochester,...
Muscular Dystrophy Treatment Shows Promise
...sults are encouraging that effective therapies for myotonic
[muscle-stiffening] dystrophy can be developed tha...ctive therapy in humans."
According to the MDA, myotonic
dystrophy is one of nine forms of muscular dystrop...eases which primarily affect voluntary muscles.
dystrophy type 1 is the most common form of muscul...
New center to lead international muscular dystrophy research effort
...trumental to the identification of novel evidence-based intervention strategies.
URMCs Department of Neurology is home to the National Registry of myotonic
and FSHD Patients and Family Members. The registry which is funded by the National Institutes of Health (NIH) will enable Fields Center researchers...
Researcher Studying Disease That Cripples Newborns
...shocked to learn their child has type 1 congenital myotonic
dystrophy (CDM1), a progressive and crippling gene...instances, mothers dont know that they have type 1 myotonic
dystrophy (DM1) a common form of muscular dystrop...ystem and winner of the 2007 Rachel Fund Award for myotonic
dystrophy (DM) research.
DM1 is the natio...
Myotonic in Medical Technology
Insmed Receives FDA Orphan Drug Designation for IPLEX(TM) in the Treatment of Myotonic Muscular Dystrophy
RICHMOND, Va., Dec. 18 /PRNewswire-FirstCall/ -- Insmed Inc. (Nasdaq:
INSM), a developer of follow-on biologics and biopharmaceuticals, today
announced that the Food and Drug Administration (FDA) has granted Orphan
Drug Designation for IPLEX(TM) for the treatment of Myotonic
Insmed Provides Update on Follow-On Biologics and IPLEX(TM) Programs
... due to severe primary IGF-I deficiency, is
currently being investigated in myotonic
Muscular Dystrophy (MMD) and
Amyotrophic Lateral Sclerosis (ALS), or Lou Ge...vestigated for various other indications with unmet medical needs
Muscular Dystrophy (MMD) and Amyotrophic Lateral
Insmed Releases Positive Results from Iplex Phase II HIV-Associated
Adipose Redistribution Syndrome Clinical Study
...predominant binding protein IGFBP-3 (rhIGFBP-3). The drug
is also being investigated for various other indications with unmet
medical needs, including myotonic
muscular dystrophy, retinopathy
of prematurity and amyotrophic lateral sclerosis (also known as ALS
or Lou Gehrig's disease).
About the University o...
Insmed Announces Promising Results From IPLEX Phase II Myotonic
Muscular Dystrophy Clinical Study
...of the company's drug, IPLEX(TM), in
patients with myotonic
muscular dystrophy (DM1).
Preliminary results of ...he results from this preliminary
study of IPLEX in myotonic
dystrophy. The results not only indicate
IPLEX was... on the preliminary
results of this study.
Insmed Inc. Welcomes Studies Linking IGFBP-3 to Prevention of
Blindness in Premature Infants
...been approved by the U.S. Food and Drug
Administration for the treatment of a severe growth disorder and is
currently being studied as a treatment for myotonic
dystrophy, HIV-associated adipose redistribution syndrome and
retinopathy of prematurity (ROP). IPLEX(TM) is the combination of
IGFBP-3 with ...
Myotonic in Medical Dictionary
dystrophy is a chronic, slowly progressing, highl...l information, assistance ... People who have myotonic
dystrophy have muscle wasting and weakness in their ... Signs and symptoms of myotonic
dystrophy usually develop when a person is in h...
...te Children's Hospital provides world class care and services to patients. ... Some common causes of hypotonia are Down syndrome, myasthenia gravis, myotonic
... List of 72 disease causes of Hypotonia in children, patient stories, diagnostic guides. Diagnostic checklist, medical tests, doctor questi...
Myotonic in Biological News
UVA reports surprising findings related to myotonic muscular dystrophy
CHARLOTTESVILLE, VA (Dec. 17, 2007) New research from the University of Virginia Health System shows that, in cases of Type 1 myotonic
muscular dystrophy (DM1), a well known heart protein does several surprising things. DM1 is the most common form of muscular dystrophy in adults and affects appr...
Researchers gain insight into mechanism underlying Huntington's
...ire DNA synthesis, DNA replication and/or DNA repair must be involved.
Two key TNRs, CAG and CTG repeats associated with Huntington's disease and myotonic
dystrophy, respectively tend to form hairpin structures via strand slippage in the newly synthesized or "nicked" DNA strand during DNA synthesis asso...
Small molecules might block mutant protein production in Huntington's disease
...s. CAG repeats are involved in several other neurodegenerative diseases, including Fragile X syndrome, the most common form of mental retardation, and myotonic
While these genes are best known for the devastating effects of their mutated forms, their normal forms are essential for embryonic deve...
American Chemical Society's Weekly PressPac -- Nov. 19, 2008
...ing a critical first step toward development of a long-sought drug to treat myotonic
muscular dystrophy (MMD), the most common form of muscular dystrophy in adu...epeat RNA-MBNL1 Interaction In Vitro: Discovery of Lead Compounds Targeting myotonic
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Benjamin L. ...
Toxic molecule may cause most common type of muscular dystrophy
...RNA (ribonucleic acid) in muscle cells can reverse myotonic
dystrophy, the most common type of muscular dystro....
About 40,000 people in the United States have myotonic
muscular dystrophy (MMD). The disease can cause a ... To prove the theory that toxic RNA is involved in myotonic
muscular dystrophy, a research team led by Dr. Man...
Myotonic in Biological Technology
Insmed Announces Results of IPLEX(TM) Phase II Trial in Myotonic Muscular Dystrophy
- Insmed to Evaluate Potential Initiation of Phase II Trial for IPLEX(TM) in MMD Patients with Severe Insulin Resistance -
RICHMOND, Va. , June 25 /PRNewswire-FirstCall/ -- Insmed Inc. (Nasdaq: INSM ), a biopharmaceutical company, today announced results from its exploratory U.S. Phase II ...
Insmed Announces First Quarter 2009 Financial Results
... approximately $125 million.
Continue to expect preliminary Phase 2 data in myotonic
Muscular Dystrophy (MMD) in the second quarter of 2009;
The FDA announced that it would allow Insmed to provide access to IPLEX(TM) for in...
Insmed Announces Fourth Quarter and Full-Year 2008 Financial Results
Completed the Phase 2 trial of IPLEX(TM) in myotonic
Muscular Dystrophy (MMD), and expect preliminary data in the second quarter of 2009;
Completed an external assessment of the total market ...
Insmed Announces Financial Results for Third Quarter and Nine-Months Ended September 30, 2008
...EAP to treat patients with ALS in Italy and the
grant receipt of $1.0 million from the Muscular Dystrophy Association
supporting the IPLEX(TM) Phase 2 myotonic
Muscular Dystrophy ("MMD") trial.
The net loss for the third quarter of 2008 was $2.2 million or $0.02
per share, compared with a net loss of $3.9 m...
Insmed and Premacure Cite Study Results Demonstrating Potential Effectiveness of IPLEX(TM) in Preventing Blindness in Premature Infants
...s serve as further evidence of the effectiveness of IPLEX(TM) in
potentially treating some of the most under-served therapeutic populations,
Muscular Dystrophy and Amyotrophic Lateral Sclerosis."
Clinical Study Results
Low levels of IGF-I are known to contribute to the pathogenesis of R...
Insmed Announces Financial Results for Second Quarter and Six-Months Ended June 30, 2008
...s strategic financial advisor to evaluate and focus the Company's strategic initiatives. -- IPLEX(TM) -- The Phase 2 trial of IPLEX(TM) in myotonic
Muscular Dystrophy ("MMD"), which was initiated in December 2007, has seen a strong patient and physician interest and is now fully enro...
Insmed CEO to Present at Drug Discovery & Development Conference
... CEO Geoffrey Allan Will Discuss Company's IPLEX(TM) Program in myotonic
RICHMOND, Va., July 25 /PRNe... He will present Insmed's progress
with its Phase II trial for IPLEX(TM) in myotonic
Muscular Dystrophy. The
trial, which has seen strong patient and physician ...
Insmed Announces First Quarter 2008 Financial Results
...ved Investigational Review Board approvals and are actively recruiting trial participants in the Phase 2 clinical trial for IPLEX(TM) in myotonic
Muscular Dystrophy ("MMD"). The trial presently has 50% of the required 60 patients enrolled and: -- Received $2.3 million in cost ...
Insmed Announces Fourth Quarter and Full-Year 2007 Financial Results
...he full-year 2007; -- Announced positive results from a Phase 2 investigator-sponsored study of IPLEX(TM) in six patients with myotonic
muscular dystrophy ("MMD") that met the primary study endpoints of being safe and well-tolerated, and indicated improvements in pati...
Insmed Awarded $2.1 Million by Muscular Dystrophy Association
Phase 3 enabling trial in patients with myotonic
Muscular Dystrophy (MMD).
Insmed, a development ... more of several symptoms
commonly associated with myotonic
Muscular Dystrophy. Furthermore, patients
professional and public health education.
Myotonic in Biological Definition
...tic ovary syndrome , chronic pancreatitis , hepatic steatosis (fatty liver), cystic fibrosis , several mitochondrial neuropathies and myopathies, myotonic
dystrophy , Friedreich's ataxia , some of the inherited forms of neonatal hyperinsulinism and many others. Risk of diabetes is higher with chronic u...
Myotonic in Biological Dictionary
Triplet repeat expansion
...xpansion may result in an unusual ... Triplet repeat expansion in myotonic
dystrophy alters the adjacent chromatin structure. ... is caused by an e...ar ... Recently, the CTG repeat expansion in the DMPK gene causing myotonic
dystrophy ... in two patients by triplet repeat -primed PCR (tpPCR) usin...