Search Results for "Ullrich Muscular Dystrophy Life Expectancy"

15:15 EDT 1st July 2015 | 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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Muscular Dystrophy treatment pipeline offers hope for improved life expectancy, says GBI Research

GBI Research reports on the highly diverse and innovative pipeline for Duchenne Muscular Dystrophy and Becker Muscular Dystrophy treatment... The post Muscular Dystrophy treatment pipeline offers hope...

Muscular dystrophy drug pipeline offers hope for improved life expectancy

The highly diverse and innovative pipeline for Duchenne muscular dystrophy (DMD) and Becker muscular…

Researchers report new figures on two muscular dystrophy disorders

Researchers in public health have reported in the first broad study in the United States the frequency of two muscle-weakness disorders that strike mostly boys: Duchenne muscular dystrophy and Becker ...

Frontier Pharma: Duchenne Muscular Dystrophy and Becker Muscular Dystrophy - Identifying and Commercializing First-in-Class Innovation

Frontier Pharma: Duchenne Muscular Dystrophy and Becker Muscular Dystrophy - Identifying and Commercializing First-in-Class Innovation Summary Highly Innovative and Diverse Pipeline The Duchenne Muscu...

EpiCast Report: Duchenne Muscular Dystrophy - Epidemiology Forecast to 2023

EpiCast Report: Duchenne Muscular Dystrophy - Epidemiology Forecast to 2023 Summary Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are rare progressive muscle wasting disorders....

Potential Treatment for Most Common Form of Muscular Dystrophy

A doctor who was one of the discoverers of the gene responsible for myotonic muscular dystrophy has now identified a therapeutic that could slow progression of muscle damage and muscle dysfunction ass...

New figures on two muscular dystrophy disorders

Public health researchers report the freqency of two muscle-weakness disorders that strike mostly boys: Duchenne muscular dystrophy and Becker muscular dystrophy. The team found that about 1 in 5,000 ...

Researchers report new figures on 2 muscular dystrophy disorders

(University of Iowa) Public health researchers report the freqency of two muscle-weakness disorders that strike mostly boys: Duchenne muscular dystrophy and Becker muscular dystrophy. The team found t...

Matching PubMed Articles

Facioscapulohumeral muscular dystrophy: Report of seven patients.

Facioscapulohumeral muscular dystrophy is the third most common muscular dystrophy with an estimated prevalence of 1 per 20.000 and a normal life expectancy in the majority of patients. However, appro...

Facioscapulohumeral muscular dystrophy and limb-girdle muscular dystrophy: "Double trouble" overlapping syndrome?

Sleep quality of mother-caregivers of Duchenne muscular dystrophy patients.

Sleep disturbance is a common problem for caregivers. In general, patients with Duchenne muscular dystrophy (DMD) use noninvasive ventilation to maintain quality of life and improve survival.

Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: a randomised, double-blind, placebo-controlled trial.

Cardiomyopathy is a leading cause of death in patients with Duchenne muscular dystrophy and myocardial damage precedes decline in left ventricular systolic function. We tested the efficacy of eplereno...

Back pain in Duchenne muscular dystrophy: steroids are not always the culprit.

We report on a child with Duchenne muscular dystrophy on prolonged corticosteroid treatment who presented with back pain and was subsequently found to have a monostotic fibrous dysplasia lesion of the...

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