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dystrophy in Medical News

Scientists Reverse Muscular Dystrophy in Mice

Strategy targets genetic flaw thought to be key to major form of the disease THURSDAY, July 16 (HealthDay News) -- Scientists have found a way to interfere with a stray genetic process critical in the development and progression of a major form of muscular dystrophy. The strategy, ...

Successful Beginning for Wellstone Muscular Dystrophy Center at Boston Biomedical Research Institute

WATERTOWN, Mass., July 2 /PRNewswire-USNewswire/ -- Last week, physicians, scientists, patients and members of the biotech industry from around the world gathered for the first major meeting of a unique research center on muscular dystrophy -- the Senator Paul D. Wellstone Muscular Dystrophy Coope...

Parent Project Muscular Dystrophy Holds 15th Annual Connect Conference in Atlanta

Senator Johnny Isakson, American Football Coaches Association to Receive Awards MIDDLETOWN, Ohio, June 22 /PRNewswire-USNewswire/ -- Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United...

Clues to Muscular Dystrophy Seen in Womb

Finding could lead to earlier, more effective treatment: researchers WEDNESDAY, June 17 (HealthDay News)-- English scientists may have found a way to detect muscular dystrophy in the womb or shortly after birth, giving doctors more time to treat the disease before symptoms develop. ...

Parent Project Muscular Dystrophy Awards Two End Duchenne Grants

Researchers at University of Florida -- Gainesville , University of California -- Los Angeles to Receive $250,000 For Duchenne Muscular Dystrophy Research MIDDLETOWN, Ohio, May 5 /PRNewswire-USNewswire/ -- Patricia A. Furlong, Founding President and CEO of...

Muscular Dystrophy Association Launches New Venture Philanthropy

Will Apply VC Business Practices to Funding Research TUCSON, Ariz., April 1 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association (MDA) today announced the launch of a new initiative, MDA Venture Philanthropy (MVP), which will be a wholly owned subsidiary of MDA. A 501(c)(3) nonprofit...

First Treatment for Muscular Dystrophy in Sight: Scientists Harness Exon-Skipping in Large Animal to Successfully Treat Duchenne Muscular Dystrophy

Researchers from Children's National Medical Center and colleagues in Tokyo publish results, video of first successful trial in dogs with Duchenne muscular dystrophy Washington, DC (PRWEB) March 16, 2009 -- Genetic researchers at Children's National Medical Center and...

First treatment for muscular dystrophy in sight: Scientists successfully harness exon-skipping

WASHINGTON, DCGenetic researchers at Children's National Medical Center and the National Center of Neurology and Psychiatry in Tokyo published the results of the first successful application of "multiple exon-skipping" to curb the devastating effects of Duchenne muscular dystrophy in an animal...

Muscular dystrophy mystery solved; Mizzou scientist moves closer to MD solution

COLUMBIA, Mo. Muscular dystrophy, which affects approximately 250,000 people in the United States, occurs when damaged muscle tissue is replaced with fibrous, bony or fatty tissue and loses function. While scientists have identified one protein, dystrophin, as an important piece to curing the dis...

Stater Bros. Markets Presents $1,143,000 Check to Muscular Dystrophy Association

Since 1986, Stater Bros. has raised over $6.1 Million for MDA SAN BERNARDINO, Calif., Sept. 25 /PRNewswire/ -- Stater Bros. Markets is pleased to announce that a check for $1,143,000 was presented to the MDA during the Jerry Lewis MDA Telethon over the recent Labor Day weekend. Stater Bro...

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dystrophy in Medical Technology

NIH Funds New Wellstone Research Center for Muscular Dystrophy at Boston Biomedical Research Institute

First Wellstone center in New England and first center in the world to focus exclusively on facioscapulohumeral muscular dystrophy BOSTON, Sept. 10 /PRNewswire-USNewswire/ -- The U.S. National Institutes of Health (NIH) have awarded $9 million to launch a unique collaboration of researchers,...

PTC Therapeutics Completes Enrollment of Pivotal Trial in Muscular Dystrophy

- Ataluren designated as generic name for PTC124 - SOUTH PLAINFIELD, N.J., Feb. 3 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced that it has successfully completed patient enrollment of its pivotal clinical trial of PTC124 in patients with nonsense mutation Duchenne and Becker m...

PTC Therapeutics Announces Data Showing Six-Minute Walk Test Consistently Measures Ambulatory Function in Patients With Duchenne Muscular Dystrophy

- Findings Suggest Clinically Meaningful Primary Endpoint for Clinical Trials; Data Presented at the 13th International World Muscle Society Congress - SOUTH PLAINFIELD, N.J., Sept. 30 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced promising findings in patients with Duchenne/B...

PTC Therapeutics Announces Initiation of Phase 2b Registration-Directed Clinical Trial of PTC124 in Duchenne/Becker Muscular Dystrophy

First Registration Study of an Investigational Drug for Duchenne/Becker Muscular Dystrophy SOUTH PLAINFIELD, N.J., April 23 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), today announced the initiation of an international pivotal trial of PTC124 in patients with Duchen...

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 Muscular Dystrophy...

PTC Therapeutics Announces Additional Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy

Data Presented at the World Muscle Society International Congress SOUTH PLAINFIELD, N.J., Oct. 18 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small-molecule drugs targeting post-transcriptional con...

PTC Therapeutics Announces Pharmacokinetic and Safety Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy

- Data Presented at 36th Child Neurology Society Annual Meeting - SOUTH PLAINFIELD, N.J., Oct. 11 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development and commercialization of small-molecule drugs targeting post-transcriptional cont...

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

RICHMOND, Va.--(BUSINESS WIRE)--May 3, 2007 - Insmed Inc. (Nasdaq:INSM) today announced positive results from a Phase II investigator-sponsored study of the company's drug, IPLEX(TM), in patients with myotonic muscular dystrophy (DM1). Preliminary results of the clinical study, being conducted at ...

PTC Therapeutics Announces Additional Positive Interim Phase 2 Results of PTC124 in Duchenne Muscular Dystrophy

Data Presented at 59th American Academy of Neurology Annual Meeting BOSTON and SOUTH PLAINFIELD, N.J., May 04, 2007 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of small-molecule drugs targeting post-transcriptional control proc...

Got Pain?

...hed. Abnormal muscle function and injury can produce hand pain such complex regional pain syndrome (CRPS 1), or formally known as reflex sympathetic dystrophy (RSD) ( http://orthoinfo.aaos.org/topic.cfm?topic=A00021 ). Previous treatments included surgery and therapy. L. Andrew Koma...
dystrophy in Medical Definition

Autoimmune regulator

...e rare autoimmune syndrome Autoimmune Polyendocrinopathy Syndrome type 1 ( APS-1 ), also known as Autoimmune Polyendocrinopathy-Candidiasis-Ectodermal dystrophy (APECED). Disruption of AIRE results in the development of a range of autoimmune diseases, the most common clinical conditions in the syndrome are h...

Pathology

... Sclera and cornea Scleritis - Keratitis - Corneal ulcer - Snow blindness - Thygeson's superficial punctate keratopathy - Fuchs' dystrophy - Keratoconus - Keratoconjunctivitis sicca - Arc eye - Keratoconjunctivitis - Corneal neovascularization - Kayser-Fleischer ring - Arcus...

Muscle atrophy

...he age-related decrease in muscle function ( sarcopenia ), there are other diseases which may be caused by structural defects in the muscle ( muscular dystrophy ), or by inflammatory reactions in the body directed against muscle (the myopathies). Pathophysiology Muscle atrophy occurs by a change in...

Proteopathy

...ma Calcitonin Cardiac atrial amyloidosis Atrial natriuretic factor Pituitary prolactinoma Prolactin Hereditary lattice corneal dystrophy Keratoepithelin Cutaneous lichen amyloidosis Keratins Corneal lactoferrin amyloidosis Lactoferrin Pulmonary alveolar proteinos...
dystrophy in Medical Dictionary

Reflex Sympathetic Dystrophy

User-edited article about CRPS, a chronic progressive disease characterized by severe pain, swelling, and changes in the skin. Also commonly known as reflex ... Also known as Reflex Sympathetic Dystrophy (RSD), the CRPS is an uncommon nerve disorder with intense pain usually a...

Myotonic Dystrophy

Myotonic dystrophy is a chronic, slowly progressing, highly variable inherited multisystemic disease that can manifest at any age from birth to old age. It is characterized by wasting of the muscles, posterior subcapsular... Provides description, genetic changes, inheritance factors, ...

Muscular dystrophy

Muscular dystrophy (abbreviated MD) refers to a group of genetic, hereditary ... The best-known type, Duchenne muscular dystrophy (DMD), is inherited in an ... Includes disease information, treatment, prognosis, and clinical trials. From the National Institute of Neurological...

Tibial

...nd sensation to the ... Dysfunction of a single nerve group, such as the tibial nerve, is classified as mononeuropathy. ... Tibial muscular dystrophy is a condition that affects the muscles at the front of the lower leg. ... very slowly in people with tibial muscular dystrophy. ... Tibial ...

Muscular Dystrophies

... Muscular dystrophy (abbreviated MD) refers to a group of genetic, h...y ... The best-known type, Duchenne muscular dystrophy (DMD), is inherited in an ... Duchenne muscular dystrophy (DMD) is a severe recessive X-linked form of mus...

Laceration

...RATION . Signs and Symptoms. The patient with a corneal laceration has ... Corneal Laceration . Salzmann's Nodular Degeneration. Fuchs' Endothelial dystrophy ... A laceration is a wound caused by a sharp object producing edges that may be ... The four goals of laceration repair are to stop bleeding,...

ALS

...sis for both patients and health professionals. From the Muscular ... The ALS ( Amyotrophic lateral sclerosis ) Division of the MDA (Muscular dystrophy Association) ... Everyday Life With ALS : A Practical Guide " Meals for Easy ... ALSforums is an all-volunteer support group provided free of c...
dystrophy in Biological News

Muscular dystrophy diagnosis delayed almost 2.5 years in boys

Boys show signs of Duchenne Muscular Dystrophy (DMD) for 2 years before they obtain a diagnosis and disease-specific treatment, about the same length of delay children have endured for the past 20 years despite advances in genetic testing and treatment. A simple and inexpensive blood test for any...

Scientist clears hurdles for muscular dystrophy therapy

COLUMBIA, Mo. Approximately 250,000 people in the United States have some form of muscular dystrophy. Duchenne muscular dystrophy (DMD) is the most common type of the disease, predominantly affecting males. Boys with DMD will lose the ability to walk by their teens and typically die before the ag...

New therapy substitutes missing protein in those with muscular dystrophy

Researchers at the University of Minnesota Medical School have discovered a new therapy that shows potential to treat people with Duchenne muscular dystrophy, a fatal disease and the most common form of muscular dystrophy in children. In the mouse model, researchers were able to substitute for ...

March of Dimes awards $250,000 prize to scientists unraveling the causes of muscular dystrophy

BALTIMORE, MAY 3, 2009 Two scientists whose work has led to new and better ways to diagnose and potentially treat muscular dystrophy have been chosen to receive the 2009 March of Dimes Prize in Developmental Biology. Kevin P. Campbell, Ph.D. and Louis M. Kunkel, Ph.D., will share the 2009 Mar...

While focusing on heart disease, researchers discover new tactic against fatal muscular dystrophy

NEW YORK (Feb. 8, 2009) Based on a striking similarity between heart disease and Duchenne muscular dystrophy, researchers at Columbia University Medical Center have discovered that a new class of experimental drugs for heart failure may also help treat the fatal muscular disorder. At first gla...

Cell 'anchors' required to prevent muscular dystrophy

DURHAM, N.C. A protein that was first identified for playing a key role in regulating normal heart rhythms also appears to be significant in helping muscle cells survive the forces of muscle contraction. The clue was a laboratory mouse that seemed to have a form of muscular dystrophy. A group...

Potential therapy for congenital muscular dystrophy

Reno, Nevada Current research suggests laminin, a protein that helps cells stick together, may lead to enhanced muscle repair in muscular dystrophy. The related report by Rooney et al, "Laminin-111 restores regenerative capacity in a mouse model for alpha 7 integrin congenital myopathy," appears...

Researchers discover molecular basis of a form of muscular dystrophy

A team of French and German researchers report in the May 2008 print issue of The FASEB Journal ( http://www.fasebj.org ) that people with limb-girdle muscular dystrophy are missing a protein called c-FLIP, which the body uses to prevent the loss of muscle tissue. By targeting the cellular and mol...

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...

Switching genes to overdrive improves muscular dystrophy symptoms in mice

Scientists at Dana-Farber Cancer Institute have shown in a laboratory study that revving up a crucial set of muscle genes counteracts the damage caused by a form of muscular dystrophy. Reporting in the April 1 issue of Genes and Development, the researchers demonstrated that manipulating a geneti...

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dystrophy in Biological Technology

Insmed Awarded $2.1 Million by Muscular Dystrophy Association

RICHMOND, Va., Dec. 12 /PRNewswire-FirstCall/ -- Insmed Inc. (Nasdaq: INSM), today announced that it has been awarded a grant of $2,087,325 from the Muscular Dystrophy Association (MDA). The grant is expected to cover a substantial portion of the external costs associated with Insmed's 24-week...

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 ...

AMT Accesses Technology for Treatment of Duchenne Muscular Dystrophy

AMSTERDAM, The Netherlands, May 22 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it obtained a license from La Sapienza University in Rome, Italy, to their advanced small nuclear RNA (snRNA)-based e...

Repligen Reports First Quarter Fiscal Year 2010 Financial Results

... of $2,588,000, consisting primarily of royalty payments from Bristol-Myers Squibb on the U.S. sales of Orencia(R) and grant revenue from the Muscular dystrophy Association. Operating expenses for the first quarter of fiscal year 2010 were $6,489,000 compared to $5,702,000 for the same period in fiscal ...

Bay Area Youth to Represent ALS Patients at Giants' and Angels' Baseball Games on July 4th

...gue Baseball's "4ALS Awareness" campaign. MLB is working with four leading organizations -- The ALS Association, ALS TDI, Augie's Quest (the Muscular dystrophy Association's ALS research initiative) and Project A.L.S. -- whose primary goal is to find a cure for ALS. The initiative will culminate on July 4, 20...

PTC Therapeutics Receives $2.6 Million in Government Grants

... of ataluren (PTC124(TM)) in nonsense mutation Duchenne and Becker muscular dystrophy (nmDMD/BMD). (Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PT...osis Foundation), FDA's Office of Orphan Products Development, the Muscular dystrophy Association, the Parent Project Muscular Dystrophy, and the National Center...

Assessment of safety and efficacy of human embryonic stem cell therapy

... animal model. Importantly, we also showed that vision can be rescued in a mouse model of Stargardt's disease, which is a form of early-onset macular dystrophy that leads to blindness despite an intact Bruch's membrane (critical for RPE cell attachment and potential clinical efficacy). "We have worked wi...

Repligen Reports Fourth Quarter and Fiscal Year 2009 Financial Results

...000, comprised primarily of royalty payments from Bristol-Myers Squibb on the U.S. sales of Orencia(R) (abatacept) and grant revenue from the Muscular dystrophy Association. Operating expenses for fiscal year 2009 were $25,482,000 compared to $23,574,000, exclusive of the net gain of $40,170,000 from the ...

MEDomics Announces MitoDx(TM), the First NextGen Mitochondrial Genome Diagnostic Test

...ations in mitochondrial DNA is now thought to be common in both adults and children. In childhood, mitochondrial disease is more common than muscular dystrophy or cancer. Most mitochondrial disease may go undiagnosed because a primary care physician does not suspect the disease or because the causative mutati...

Insmed Announces First Quarter 2009 Financial Results

...5 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 investigational use i...
dystrophy in Biological Definition

Diabetes mellitus

... 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 use of seve...

Gene therapy

...g to introduce genes straight into human cells, focusing on diseases caused by single-gene defects, such as cystic fibrosis , hemophilia , muscular dystrophy and sickle cell anemia . However, this has been much harder than modifying simple bacteria, primarily because of the problems involved in carrying l...

Muscle

... or decreased muscle tone, respectively. The myopathies are all diseases affecting the muscle itself, rather than its nervous control. Muscular dystrophy is a large group of diseases, many of them hereditary, where the muscle integrity is disrupted. It leads to progressive loss of strength, high depend...

Stem cell

...ferentiate to form specific types of brain cells. This could lead to new treatments and cures for diseases like Lou Gehrig's disease , muscular dystrophy , and spinal cord injuries. Researchers at the University of Wisconsin-Madison coaxed human embryonic stem cells into becoming neural stem cel...

X chromosome

...agile X syndrome hemophilia incontinentia pigmenti Lesch-Nyhan syndrome Menkes syndrome Duchenne and Becker's muscular dystrophy nonsyndromic deafness and X-linked nonsyndromic deafness Rett syndrome spinal and bulbar muscular atrophy X-linked severe combined i...
dystrophy in Biological Dictionary

Triplet repeat expansion

...ese fall into two groups: (1) those that... ... dystrophy is caused by expansion of a GCG repeat that pl... ... Triplet repeat expansion in myotonic dystrophy alters the adjacent chromatin structure. ... is ...t expansion in the DMPK gene causing myotonic dystrophy ... in two patients by triplet repeat -primed P...

Sarcoplasmic reticulum

... rabbit. ... The purified ATPase of sarcoplasmic reticulum exists as particles with minimum ... Sarcoplasmic Reticulum in Duchenne Muscular dystrophy ... Sarcoplasmic reticulum fragments isolated from dog cardiac muscle possess a ... The sarcoplasmic reticulum preparation produced gluc...
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