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Myotonic in Medical News

Scientists Reverse Muscular Dystrophy in Mice

... you can impact the molecular underpinnings of myotonic dystrophy, which is an important step forward in ... causes," she said. According to the MDA, myotonic (muscle-stiffening) dystrophy is one of nine ... which primarily affect voluntary muscles. myotonic dystrophy type 1 is the most common form of ...

Scientists exploring new compounds to target muscular dystrophy

... that targets the wayward RNA at the root of myotonic muscular dystrophy," said Benjamin Miller, Ph.D., ... 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 ...

Muscular Dystrophy Treatment Shows Promise

... are encouraging that effective therapies for myotonic [muscle-stiffening] dystrophy can be developed ... therapy in humans." According to the MDA, myotonic dystrophy is one of nine forms of muscular ... which primarily affect voluntary muscles. myotonic dystrophy type 1 is the most common form of ...

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 ... splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy AUTHOR CONTACT: Charles A. Thornton University of ...

Insmed Provides Update on Supply of IPLEX(TM)

... 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 ...

Muscular Dystrophy Association Launches New Venture Philanthropy

... 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 ...

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 Muscular Dystrophy. About Insmed Insmed Inc. is a biopharmaceutical company with unique protein process development and manufacturing experience ...

New center to lead international muscular dystrophy research effort

... 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 ...

Researcher Studying Disease That Cripples Newborns

... to learn their child has type 1 congenital myotonic dystrophy (CDM1), a progressive and crippling ... mothers dont know that they have type 1 myotonic dystrophy (DM1) a common form of muscular ... and winner of the 2007 Rachel Fund Award for myotonic dystrophy (DM) research. DM1 is the ...
Myotonic in Medical Technology

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 ... for various other indications with unmet medical needs including myotonic Muscular Dystrophy (MMD) and Amyotrophic Lateral Sclerosis (ALS). About ...

Insmed Receives FDA Orphan Drug Designation for IPLEX(TM) in the Treatment of Myotonic Muscular Dystrophy

... today announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation for IPLEX(TM) for the treatment of myotonic Muscular Dystrophy (MMD). Insmed is currently conducting a 24-week Phase III enabling trial for IPLEX(TM) in MMD, and recently was awarded a grant ...

Insmed Announces Promising Results From IPLEX Phase II Myotonic Muscular Dystrophy Clinical Study

... the company's drug, IPLEX(TM), in patients with myotonic muscular dystrophy (DM1). Preliminary results of ... results from this preliminary study of IPLEX in myotonic dystrophy. The results not only indicate IPLEX ... on the preliminary results of this study. About myotonic Muscular Dystrophy ...

Insmed Releases Positive Results from Iplex Phase II HIV-Associated Adipose Redistribution Syndrome Clinical Study

... 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 ...

Insmed Inc. Welcomes Studies Linking IGFBP-3 to Prevention of Blindness in Premature Infants

... 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 muscular dystrophy, HIV-associated adipose redistribution syndrome and retinopathy of prematurity (ROP). IPLEX(TM) is the combination of IGFBP-3 with ...
Myotonic in Medical Dictionary

Myotonic Dystrophy

... myotonic dystrophy , type 2: An inherited disorder of the ... ( cataracts ), cardiac abnormalities, ... myotonic dystrophy (DM), also called dystrophia ... and females approximately equally. What is myotonic dystrophy ? An example of an autosomal dominant ...

Stuttering

... no cure for stammering but many therapies can make a difference ... myotonic dystrophy is due to a trinucleotide repeat (a " stuttering " sequence of three bases) in the DNA. The myotonic dystrophy gene (called DM1), found on chromosome 19q13.3, codes for a ...

Galactose

... In most people the lactase enzyme levels decrease with age, so a person actually "grows into" lactose intolerance . These disorders include myotonic dystrophy , the most common form of muscular dystrophy in adults, and galactose mia , a rare disorder that involves elevated levels of the sugar ...

Galactosemia

... , cephalo hematoma , polycythemia , G-6-P-D deficiency , and congenital rubella , syphilis , toxoplasmosis , ... These disorders include myotonic dystrophy , the most common form of muscular dystrophy in adults, and galactosemia , a rare disorder that involves elevated levels of the sugar ...

Myotonia congenita

... However, many patients with myotonia do experience cramp ing from exercise . Symptoms of myotonia are often worse in the cold . Myotonias include myotonic dystrophy , myotonia congenita , para myotonia congenita , and neuromyotonia. ...

Neurologic deficit

... s Muscle defects Connective tissue and skeletal defects Fetal crowding or fetal constraint Maternal neuromuscular disease s -- myotonic dystrophy and myasthenia gravis . Significant visual deficits are found in 10-50 percent of survivors, and other major neurologic deficit s in ...

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Myotonic in Biological News

American Chemical Society's Weekly PressPac -- Nov. 19, 2008

... 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 ... RNA-MBNL1 Interaction In Vitro: Discovery of Lead Compounds Targeting myotonic Dystrophy (DM1)" DOWNLOAD FULL TEXT ARTICLE CONTACT: Benjamin L. ...

UVA reports surprising findings related to myotonic muscular dystrophy

... the University of Virginia Health System shows that, in cases of Type 1 myotonic muscular dystrophy (DM1), a well known heart protein does several ... diagnostic test to follow a patients response to potential therapies. myotonic muscular dystrophy is recognized as the first example of a disease caused ...

Researchers gain insight into mechanism underlying Huntington's

... 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 ...

Small molecules might block mutant protein production in Huntington's disease

... CAG repeats are involved in several other neurodegenerative diseases, including Fragile X syndrome, the most common form of mental retardation, and myotonic dystrophy. While these genes are best known for the devastating effects of their mutated forms, their normal forms are essential for embryonic ...

Toxic molecule may cause most common type of muscular dystrophy

... (ribonucleic acid) in muscle cells can reverse myotonic dystrophy, the most common type of muscular ... 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. ...
Myotonic in Biological Technology

Insmed Announces Results of IPLEX(TM) Phase II Trial in Myotonic Muscular Dystrophy

... IPLEX(TM) (mecasermin rinfabate) in patients with myotonic muscular dystrophy ("MMD"). The randomized, ... advisor, RBC Capital Markets." About myotonic Muscular Dystrophy myotonic muscular dystrophy is a genetic disorder ...

Insmed Awarded $2.1 Million by Muscular Dystrophy Association

... 24-week 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, ... and public health education. About myotonic Muscular Dystrophy ...

Insmed CEO to Present at Drug Discovery & Development Conference

... CEO Geoffrey Allan Will Discuss Company's IPLEX(TM) Program in myotonic Muscular Dystrophy RICHMOND, Va., July 25 ... 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 2009 Financial Results

... approximately $125 million. IPLEX(TM) 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 ...

Insmed Announces Fourth Quarter and Full-Year 2008 Financial Results

... 2009. IPLEX(TM) 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

... 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 ...

Insmed and Premacure Cite Study Results Demonstrating Potential Effectiveness of IPLEX(TM) in Preventing Blindness in Premature Infants

... serve as further evidence of the effectiveness of IPLEX(TM) in potentially treating some of the most under-served therapeutic populations, including myotonic Muscular Dystrophy and Amyotrophic Lateral Sclerosis." Clinical Study Results Low levels of IGF-I are known to contribute to the pathogenesis of ...

Insmed Announces Financial Results for Second Quarter and Six-Months Ended June 30, 2008

... 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 ...

Insmed Announces First Quarter 2008 Financial Results

... 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

... 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 ...
Myotonic in Biological Definition

Diabetes mellitus

... 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 ...
Myotonic in Biological Dictionary

Triplet repeat expansion

... may result in an unusual ... Triplet repeat expansion in myotonic dystrophy alters the adjacent chromatin structure. ... is caused by an ... ... Recently, the CTG repeat expansion in the DMPK gene causing myotonic dystrophy ... in two patients by triplet repeat -primed PCR (tpPCR) ...
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