Search Results for "Ullrich Muscular Dystrophy Life Expectancy"

14:01 EDT 28th April 2017 | BioPortfolio

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Duchenne muscular dystrophy - DMD

Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene...

Exon Skipping in development for DMD

The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small sequences of genetic code which, via an intermediate step involving RNA, lead to the assembly of sections...

Spinal Muscular Atrophies

Progressive muscular atrophy

Orphan Indications

Examples of Ophan Drug Indications include: Anaemia, sickle cell Cystic fibrosis (CF) Duchenne muscular dystrophy Glioma Graft vs host disease (GvHD) Hepatoma, liver cancer Hodgkin Lymphoma ...

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Not all Europeans receive the same care for Duchenne muscular dystrophy

(IOS Press) Duchenne muscular dystrophy (DMD), a progressive muscle disease affecting one in 3,800-6,300 live male births and leads to ambulatory loss, respiratory problems, cardiomyopathy, and early ...

In need of complex care, a Syrian child gets a second chance at a US hospital

Jude Almkhlef's family fled Syria in 2013. Now the 9-year-old, who has Ullrich congenital muscular dystrophy, is making great progress at Boston Children's.

Patients' Right-To-Try Should Not Be A License To Fleece Them

  In this Friday, June 5, 2015 photo, Gabe Griffin, a 10-year-old with Duchenne Muscular Dystrophy, walks on a basketball court while playing with his cousins in Birmingham, Ala. Duchenne Muscula...

Study identifies potential drug targets for muscular dystrophy treatments

Myotonic dystrophy type I (MD1) is a common form of muscular dystrophy associated with muscle wasting, weakness, and myotonia. These symptoms are linked to the accumulation of toxic gene transcripts i...

Genetic study identifies new type of muscular dystrophy

Scientists have identified a unique form of congenital muscular dystrophy, characterised by short stature, intellectual disabilities and cataracts...

Fructose Could Help Enhance Muscular Dystrophy Therapy

NewsProgressive muscle degeneration and weakness characterize Duchenne muscular dystrophy.

Drug pair slows progression of muscular dystrophy in animal models

A pair of protein-inhibiting compounds is effective at slowing the progression of a form of muscular dystrophy in animal models, a group of University of Florida Health researchers has found. The comp...

aTyr Pharma Presents Analyses of Resolaris Phase 1b/2 Trial in Patients with Limb Girdle Muscular Dystrophy 2B and Facioscapulohumeral Muscular Dystrophy at the American Academy of Neurology 69th Annual Meeting

- Resolaris Demonstrated Favorable Safety Profile and Promising Signals of Clinical Activity - - Resolaris has FDA Fast Track and Orphan Drug Designation for Limb Girdle Muscular Dystrophy 2B (LG...

Matching PubMed Articles

New Survival Target for Duchenne Muscular Dystrophy.

We report a patient with a typical phenotype and clinical history of Duchenne muscular dystrophy who is currently 53 years old. Because of improvements in cardiopulmonary care, there has been a great ...

Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial.

In Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), interventions reducing the progression of myocardial disease could affect survival.

Gastrointestinal Dysfunction in Patients with Duchenne Muscular Dystrophy.

In adult patients with Duchenne muscular dystrophy (DMD) life-threatening constipation has been reported. Since gastrointestinal function in DMD has not been rigorously studied we investigated objecti...

Trends in Working Life Expectancy in Europe.

The aim of the article is to analyze past and present developments of working life expectancy (WLE) at age 50 by age, sex, and education in Europe. WLE is also compared with life expectancy (LE) and h...

Upper limb functional assessment scale for children with Duchenne muscular dystrophy and Spinal muscular atrophy.

Duchenne muscular dystrophy (DMD) and Spinal muscular atrophy (SMA) causes significant disability and progressive functional impairment. Readily available instruments that assess functionality, especi...

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