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Search Results for "Ullrich Muscular Dystrophy Life Expectancy"

15:31 EDT 17th August 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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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...

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...

Gene therapy: Microdystrophin restores muscle strength in Duchenne muscular dystrophy

(AFM-Téléthon) Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal Holloway) demonstrated the efficacy of an innovative gene therapy...

Microdystrophin restores muscle strength in Duchenne muscular dystrophy

Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR) and the Royal Holloway University of London demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne mu...

Rise in life expectancy has stalled since 2010, research shows – The Guardian

The GuardianRise in life expectancy has stalled since 2010, research showsThe GuardianSir Michael Marmot says the needs of the ageing population will place the NHS and social care under strain in the ...

Gene-editing alternative corrects Duchenne muscular dystrophy

Using the new gene-editing enzyme CRISPR-Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab.

[Comment] Treatment of Duchenne muscular dystrophy: first small steps

Duchenne muscular dystrophy (DMD) is a progressive and life-limiting X-linked recessive disorder caused by mutations in the DMD gene that result in reduced or absent dystrophin production. Dystrophin ...

Matching PubMed Articles

Translation and validation of the Life Satisfaction Index for Adolescents scale with neuromuscular disorders: LSI-A Brazil.

To validate the Life Satisfaction Index for Adolescents (LSI-A) scale, parent version and patient version, for Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA) and limb-girdle muscular...

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 ...

Parents' Reactions to the Diagnosis of Duchenne Muscular Dystrophy: Associations Between Resolution, Family Functioning, and Child Behavior Problems.

Duchenne muscular dystrophy (DMD) is the most frequent inherited form of muscular dystrophy during childhood. DMD is a severe and progressive disease. Children initially have no symptoms, but the diag...

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.

Facioscapulohumeral Muscular Dystrophy.

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

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