Search Results for "Ullrich Muscular Dystrophy Life Expectancy"

03:05 EDT 26th June 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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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.

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...

Catabasis Pharmaceuticals to Present at the 2017 Muscular Dystrophy Association Scientific Conference

Monday, March 13th 2017 at 12:00pm UTC CAMBRIDGE, Mass.–(BUSINESS WIRE)– Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it ...

Company 'Pauses' Launch of $89,000 Muscular Dystrophy Drug

A drugmaker fiven FDA approval to market a generic drug for muscular dystrophy has 'paused' rollout after an outcry over its $89,000-a-year price tag.

Researchers repurpose former experimental cancer therapy to treat muscular dystrophy

Researchers at the National Institutes of Health's National Center for Advancing Translational Sciences (NCATS) and the University of Nevada, Reno School of Medicine (UNR Med) have demonstrated that a...

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...

Facioscapulohumeral Muscular Dystrophy.

This article describes the clinical characteristics, diagnosis, molecular pathogenesis, and treatment of facioscapulohumeral muscular dystrophy (FSHD).

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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