Search Results for "Ullrich Muscular Dystrophy Life Expectancy"

07:54 EDT 30th August 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…

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...

FDA issues draft guidance on DMD drug development

FDA issued draft guidance to advise companies developing candidates to treat X-linked Duchenne muscular dystrophy (DMD) and related diseases, including Becker muscular dystrophy, DMD-associated dilate...

OpportunityAnalyzer: Duchenne Muscular Dystrophy - Opportunity and Market Analysis to 2019

OpportunityAnalyzer: Duchenne Muscular Dystrophy - Opportunity and Market Analysis to 2019 Summary Duchenne muscular dystrophy (DMD) is a rare genetic disorder affecting approximately 1 in 3,500 newbo...

Cardiac and respiratory function supported by abdominal muscles in muscular dystrophy

The muscular dystrophies are known to target various muscle groups differentially. In addition to making limb muscles weak, muscular dystrophy (MD) can also lead to decreased function of specific musc...

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...

Muscle Quantitative MR Imaging and Clustering Analysis in Patients with Facioscapulohumeral Muscular Dystrophy Type 1.

Facioscapulohumeral muscular dystrophy type 1 (FSHD1) is the third most common inherited muscular dystrophy. Considering the highly variable clinical expression and the slow disease progression, sensi...

Duchenne muscular dystrophy: current cell therapies.

Duchenne muscular dystrophy is a genetically determined X-linked disease and the most common, progressive pediatric muscle disorder. For decades, research has been conducted to find an effective thera...

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.

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