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Latest Becker's muscular dystrophy Stories

2014-07-09 16:28:13

-French ATU Cohort and Other Named Patient Programs Authorized - SOUTH PLAINFIELD, N.J., July 9, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the initiation of a reimbursed expanded access program (EAP). PTC's EAP program is intended to make Translarna(TM) (ataluren) available to patients before commercial availability in certain countries. Where mechanisms exist and in accordance with local regulations, PTC will make Translarna available to nonsense...

2014-06-25 12:30:09

Parent Project Muscular Dystrophy-led guidance seeks to address unmet clinical needs of individuals with Duchenne muscular dystrophy and accelerate development of safe and effective therapies CHICAGO, June 25, 2014 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) and a broad coalition of stakeholders today submitted the first-ever patient advocacy-initiated draft guidance for a rare disease to the U.S. Food and Drug Administration (FDA) to help accelerate development and...

2014-02-19 12:29:18

HACKENSACK, N.J., Feb. 19, 2014 /PRNewswire-USNewswire/ -- DuchenneConnect, a program of Parent Project Muscular Dystrophy (PPMD), is thrilled to announce receiving a funding award of $970,000 by the Patient-Centered Outcomes Research Institute (PCORI) to take part in developing a health data network. DuchenneConnect will be part of PCORnet: the National Patient-Centered Clinical Research Network. The DuchenneConnect network is one of 29 networks that were approved for PCORnet. The...

2014-01-24 23:02:29

Recent scientific development represents progress in FSHD research. Lexington, Mass. (PRWEB) January 24, 2014 As published online today in Human Molecular Genetics, support from the FSH Society, a patient-driven nonprofit, has enabled people with facioscapulohumeral muscular dystrophy (FSHD) to donate muscle tissue, which scientists have succeeded in grafting into mice, providing a new tool for conquering this devastating muscle-wasting disease. Among the most common forms of muscular...

2013-09-18 12:55:59

A preclinical study led by researchers at Children’s National Medical Center has found that a new oral drug shows early promise for the treatment of Duchenne muscular dystrophy (DMD). The results, published in EMBO Molecular Medicine, show that the drug, VBP15, decreases inflammation and protects and strengthens muscle without the harsh side effects linked to current treatments with glucocorticoids such as prednisone. Duchenne muscular dystrophy results in severe muscle degeneration and...

2013-07-08 12:13:59

Children with Duchenne muscular dystrophy often die as young adults from heart and breathing complications. However, scientists have been puzzled for decades by the fact that laboratory mice bearing the same genetic mutation responsible for the disease in humans display only mild symptoms and no cardiac involvement. Now, researchers at the Stanford University School of Medicine have developed a mouse model that accurately mimics the course of the disease in humans. The study is the first...

Promise In Duchenne Muscular Dystrophy Shown With Genetic Editing
2013-06-05 08:48:09

Duke University Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients. Instead of the common gene therapy approach of adding new genetic material to "override" the faulty gene, the Duke scientists have developed a way to change the existing mutated gene responsible for the disorder into a normally...


Word of the Day
tesla
  • The unit of magnetic flux density in the International System of Units, equal to the magnitude of the magnetic field vector necessary to produce a force of one newton on a charge of one coulomb moving perpendicular to the direction of the magnetic field vector with a velocity of one meter per second. It is equivalent to one weber per square meter.
This word is named for Nikola Tesla, the inventor, engineer, and futurist.