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Families of Spinal Muscular Atrophy Announces Latest Developments on Three SMA Drug Discovery Programs

... 18 /PRNewswire-USNewswire/ -- Families of Spinal muscular Atrophy has just released details of the latest ... About Families of SMA: Families of Spinal muscular Atrophy is dedicated to creating a treatment and ... Our vision is a world where Spinal muscular Atrophy is treatable and curable. The goal ...

PTC Therapeutics Completes Enrollment of Pivotal Trial in Muscular Dystrophy

... with nonsense mutation Duchenne and Becker muscular dystrophy (nmDMD/BMD). Accrual was completed two ... teams around the world." "Duchenne/Becker muscular dystrophy is a rare disorder and patients whose ... develop additional innovative therapies for the muscular dystrophy community." About DMD/BMD ...

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

... of cardiac, respiratory, circulatory and muscular capacity. Until this study, it had been unclear ... interruption from falls. The study found that the muscular deficits that are characteristic of boys with ... able to detect treatment effects on DMD-related muscular deficits. "The 6MWT evaluates the critical ...

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

... world to focus exclusively on facioscapulohumeral muscular dystrophy BOSTON, Sept. 10 ... and potential treatments for facioscapulohumeral muscular dystrophy (FSHD), a muscle weakening and ... The award will create the first Senator Wellstone muscular Dystrophy Cooperative Research Center (MD CRC) to ...

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

... for Duchenne/Becker muscular Dystrophy SOUTH PLAINFIELD, N.J., April 23 ... trial of PTC124 in patients with Duchenne/Becker muscular dystrophy (DMD/BMD) due to a nonsense mutation. ... other indications." About Duchenne and Becker muscular Dystrophy Duchenne and ...

PTC Therapeutics Announces Publication of Preclinical Data in PNAS

... of PTC124 in a preclinical model of Duchenne muscular dystrophy (DMD). PTC124 has demonstrated ... fibrosis (CF) and Duchenne muscular dystrophy (DMD). It is estimated that 10% of ... development has been supported by grants from the muscular Dystrophy Association (MDA), Cystic Fibrosis ...

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

... of genetic disorders." About Duchenne muscular Dystrophy Duchenne muscular dystrophy (DMD) is a progressive muscle disorder ... DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic ...

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

... trial of PTC124 in patients with Duchenne muscular dystrophy (DMD) due to a nonsense mutation. The ... Muscle Society meeting in Italy." About Duchenne muscular Dystrophy Duchenne muscular dystrophy (DMD) is a progressive muscle disorder ...

Boston University and The Center for Human Genetics, Inc. Announce Change in Testing Services

... developmental disorders caused by mutation of the ARX gene, Duchene's muscular dystrophy, myotonic dystrophy (DM1), Freidreich's Ataxia (FRDA), hereditary neuropathy with pressure palsies, polycystic kidney disease, spinal muscular atrophy (SMN1), spinocerebellar ataxia (SCA1, 2, 3, 6, 7 and 8) and ...

Insmed Provides Update on Follow-On Biologics and IPLEX(TM) Programs

... primary IGF-I deficiency, is currently being investigated in Myotonic muscular Dystrophy (MMD) and Amyotrophic Lateral Sclerosis (ALS), or Lou Gehrig's ... for various other indications with unmet medical needs including Myotonic muscular Dystrophy (MMD) and Amyotrophic Lateral Sclerosis (ALS). About Insmed ...

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

... and recently was awarded a grant of approximately $2.1 million from the muscular Dystrophy Association (MDA), which is expected to cover a substantial ... medical conditions, including two neuromuscular disorders, Myotonic muscular Dystrophy (MMD) and ALS (Lou Gehrig's Disease); HIV-Associated Adipose ...

ALS TDI Completes Gene Expression Profile of Lou Gehrig's Disease in Mice

... year as part of a historic three- year, $36 million partnership with the muscular Dystrophy Association (MDA) through its fast-track ALS research ... Sharon Hesterlee, Ph.D., vice president of translational research at the muscular Dystrophy Association. "As the data are analyzed and the results ...

Merz Pharmaceuticals Announce Three Studies of NT-201 - (Botulinum neurotoxin type A Free From Complexing Proteins)

... the placebo group, in 58.1% of the 120 U group and in 71.0% of the 240 U group. AEs reported most frequently were dysphagia (0% vs 19.4% vs 22.6%), muscular weakness (3.6% vs 6.5% vs 22.6%), and neck pain (3.6% vs 3.2%, vs 22.6%) for each group, respectively. Third Study - Title Repeated ...

AMT Receives EMEA Orphan Drug Designation for Acute Intermittent Porphyria

... intermediate metabolites resulting in a wide variety of problems including acute, severe abdominal pains, psychiatric, neurologic illnesses, and muscular weakness. Long-term consequences may be irreversible nerve damage, liver cancer and kidney failure. Acute porphyric attacks can be life-threatening. ...

Merz Pharmaceuticals Announces Results of Three Clinical Trials With NT-201 (Botulinum Neurotoxin Type A Free From Complexing Proteins) in Movement Disorder Patients

... U group. AEs reported most frequently for each group respectively were dysphagia (2.7% vs 12.8% vs 18.5%), neck pain (4.1% vs 6.4%, vs 14.8%), and muscular weakness (1.4% vs 6.4% vs 11.1%). Efficacy and safety of NT-201 (Botulinum neurotoxin free from complexing proteins) in Blepharospasm. ...

Torax Medical Launches Pivotal Clinical Study of New Acid Reflux Treatment

... the LINX System in patients suffering from GERD despite medical therapy. GERD is caused by a defective lower esophageal sphincter, a ring-like muscular valve that surrounds the esophagus. When defective, it allows abnormal reflux of stomach acids and bile into the esophagus. Approximately 19 million ...

AndroScience Corporation Announces Phase 2a Study Results on ASC-J9(TM) for the Treatment of Acne

... (male pattern baldness), and wound healing using topical based formulations; and prostate cancer, benign prostatic hyperplasia, and spinal bulbar muscular atrophy (Kennedy's Disease - a neuron motor disease caused by AR mutation) utilizing systemic based approaches. AndroScience Corporation ...

New Insight Into the Controls on a Go-To Enzyme

... of this broad set of responsibilities is that defective or overactive calpains have been linked to an array of disorders, including a form of muscular dystrophy, Type 2 diabetes, gastric cancers, Alzheimer's and Parkinson's diseases, cataracts, and the death of both heart muscle in heart attacks and ...

Paratek Reports Positive Phase 2 Clinical Data on its New Antibiotic PTK 0796 in Late Breaker Presentation at the 48th Annual Interscience Conference on Antimicrobial Agents and Chemotherapy

... diseases. In addition, Paratek is encouraged by early evidence of the ability of tetracycline derivatives to affect mRNA splicing, as in spinal muscular atrophy (SMA), which may also have activity in related orphan genetic disorders, such as cystic fibrosis and Duchenne Muscular Dystrophy. Paratek ...

PTC Announces Data Showing That PTC124 Causes Statistically Significant Improvements in Chloride Channel Function in Cystic Fibrosis Patients

... the United States and Canada. The development of PTC124 has also been supported by grants from the Muscular Dystrophy Association, Parent Project muscular Dystrophy, FDA's Office of Orphan Products Development, the National Center for Research Resources and notably, the Cystic Fibrosis Foundation ...

Paratek to Present Clinical Data in Late Breaker Presentation at the 48th Annual Interscience Conference on Antimicrobial Agents and Chemotherapy

... to affect mRNA splicing, as in SMA, which may also have activity in related orphan genetic disorders, such as cystic fibrosis (CF) and Duchene muscular Dystrophy. Paratek has active collaborations with Merck & Co., MerckSerono, Warner-Chilcott and FSMA to develop tetracycline-derived small ...

ALS Therapy Development Institute Partners with Clinical Centers to Establish Pathological Mechanisms of Lou Gehrig's Disease

... disease. These ongoing projects are made possible as part of a major, three-year, $18 million funding and scientific collaboration with the muscular Dystrophy Association (MDA) and its Augie's Quest Initiative, entered into at the beginning of 2007. "We're pleased to be part of this project that ...

PTC Therapeutics Announces $25 Million Award From Cystic Fibrosis Foundation Therapeutics for Development of PTC124

... process, preventing production of a functional protein. In Phase 2a clinical trials in nonsense-mutation-mediated cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD), PTC124 has demonstrated the ability to produce protein across a variety of nonsense mutation types. Across all clinical studies to ...

Halozyme Therapeutics Announces That Chemophase Meets Primary Endpoint in Phase I/IIa Clinical Trial

... the frequency of recurrence can minimize these adverse consequences. Even with the current standard of care, after transurethral resection of non- muscular invasive bladder cancer followed by currently available intravesical therapy, patients typically experience a tumor recurrence rate of 40% to 85%, of ...

PTC Therapeutics Announces Data from Additional Clinical Studies of PTC124 in Cystic Fibrosis Confirming Activity

... of a functional protein. In Phase 2a clinical trials in nonsense-mutation-mediated cystic fibrosis (CF) and in nonsense-mutation-mediated Duchenne muscular dystrophy (DMD), PTC124 has demonstrated the ability to produce functional protein across a variety of nonsense mutation types. Across all clinical ...

PTC Therapeutics Announces Data Supporting Cough Frequency as a New Outcome Measure in Evaluating Treatments for Cystic Fibrosis

... of normal stop codons. PTC124 has demonstrated pharmacodynamic proof of concept in Phase 2a clinical trials in nonsense-mutation-mediated Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF). About PTC Therapeutics Inc. PTC is a biopharmaceutical company focused on the discovery, development ...

Genzyme Genetics Announces Launch of Carrier Testing and Prenatal Diagnosis for Spinal Muscular Atrophy

... it is the first national laboratory specializing in reproductive testing to provide population carrier and prenatal diagnostic testing for spinal muscular atrophy (SMA), the most common inherited cause of infant mortality. This test will enable couples who are planning a pregnancy, or who are already ...

Recent Data Study Suggests Cortisol Could Help Alleviate Suffering for Those With Chronic Fatigue Syndrome and Fibromyalgia

... and no specific diagnostic tests are available. What is Fibromyalgia? Fibromyalgia or FM is a chronic pain condition characterized by generalized muscular pain and fatigue. Fibromyalgia typically involves pain in the muscles, ligaments and tendons and related sleep and quality of life disturbances. This ...

Do-It-Yourself Cardiac Bypass Surgery: All You Need is Walking Shoes

... artery that feeds the heart, the body responds by trying to bulk up tiny blood vessels in the heart. As these so-called collateral vessels grow more muscular and interconnected, they begin to reroute some of the blood flow around the blockage. Scientists have been trying for years to nudge collateral blood ...

Paratek Pharmaceuticals Announces the Close of $40 Million Private Placement to Finance Programs to Combat MRSA Infections

... and FSMA to develop orally available small molecule drugs for community bacterial infections, multiple sclerosis, acne & rosacea, and spinal muscular atrophy (SMA), respectively. Paratek is privately held and headquartered in Boston, Massachusetts, USA. For more information about Paratek and its ...

New Study: Pine Bark Extract Boosts Nitric Oxide (NO) Production

... blood circulation and support a lasting aerobic muscle activity during any kind of activity and enhance sports endurance by alleviating cramping and muscular pain in athletes. In short, Pycnogenol(R) is effective for enhancing and prolonging muscle performance during sport, supports muscle adaptation to ...

Promising Data from Paratek Pharmaceuticals' MAR Inhibitor Program Presented in 'Late Breaker' Session at the 47th Interscience Conference on Antimicrobial Agents and Chemotherapy

... and FSMA to develop orally available small molecule drugs for community bacterial infections, multiple sclerosis, acne & rosacea, and spinal muscular atrophy (SMA), respectively. Paratek is privately held and headquartered in Boston, Massachusetts, USA. For more information about Paratek and its ...

First Osteoporosis Study in Hip Fracture Patients Finds Once-Yearly Reclast(R) Prevents Additional Fractures and Improves Survival

... osteonecrosis of the jaw (ONJ) were seen in the study. The most common adverse events with Reclast were transient post-dose symptoms (such as fever, muscular pain, etc). About the Study The Recurrent Fracture Trial was an international, multi-center, randomized, double-blind, placebo-controlled Phase III ...

Targeting Specific Mutations Reveals Promising New Approach to Treating Genetic Disorders; Preclinical Data Published in Nature

... efficacious in a preclinical model of Duchenne muscular dystrophy (DMD). It is estimated that ... cystic fibrosis and Duchenne muscular dystrophy. The FDA has granted PTC124 ... Inc. (CFFT), Parent Project muscular Dystrophy (PPMD), FDA's Office of Orphan Products ...

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

... drug, IPLEX(TM), in patients with myotonic muscular dystrophy (DM1). Preliminary results of the ... of this study. About Myotonic muscular Dystrophy Myotonic muscular dystrophy, also known as myotonic ...

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

... trial of PTC124 in patients with Duchenne muscular dystrophy (DMD) due to a nonsense mutation. The ... genetic disorders." About Duchenne muscular Dystrophy Duchenne muscular dystrophy (DMD) is a progressive muscle disorder ...

Acceleron Pharma's ACE-031 Increases Muscle Mass and Strength in Preclinical Studies

... studies in normal mice and in a mouse model of muscular dystrophy. These studies show a ... treatment continues. In the mdx murine model of muscular dystrophy, treated animals showed increases in ... (1) neuromuscular diseases, such as ALS and muscular dystrophy, in which patients suffer from the loss ...

Accelreon Pharma's ACE-031Shown to Increase Muscle Mass in Preclinical Studies

... models of amyotrophic lateral sclerosis (ALS), muscular dystrophy, glucocorticoid-induced muscle loss and ... treatment continues. In the mdx murine model of muscular dystrophy, treated animals showed increases in ... (1) neuromuscular diseases, such as ALS and muscular dystrophy, in which patients suffer from the loss ...

The Sturdier Sex?: Study by Pittsburgh Scientists Finds Female Stem Cells Work Better

... stem cells they isolated in the lab while searching for a cure for Duchene muscular dystrophy (DMD). DMD is a genetic disease estimated to affect one in every ... the cells to stress or after cell transplantation in the animals that have muscular dystrophy. Transplantation of female cells leads to a much more ...
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