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Search Results for "Ullrich Muscular Dystrophy Life Expectancy"

14:11 EST 11th February 2016 | 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...

Progressive muscular atrophy

Spinal Muscular Atrophies

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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Merozyne – muscular dystrophy

Merozyne Therapeutics has been established to develop a treatment for MDC1A, a severe form of muscular dystrophy. Merozyne – muscular dystrophy MDC1A is a rare and severe form of muscular dystro...

Robotic arm developed for Duchenne patients

Researchers have developed the A-Gear: a robotic arm that can support the daily activities of people suffering the muscular disease Duchenne Muscular Dystrophy. They recently put the final touches to ...

Gene Editing Offers Hope for Treating Duchenne Muscular Dystrophy, Studies Find

(New York Times) – After decades of disappointingly slow progress, researchers have taken a substantial step toward a possible treatment for Duchenne muscular dystrophy with the help of a powerf...

Gene Editing Could Yield New Muscular Dystrophy Therapy

The controversial gene editing technique, Crispr-Cas9, could help create a treatment for muscular dystrophy, according to a new, early study published in the journal Science.Read more

Gene Therapy Could Yield New Muscular Dystrophy Therapy

The controversial gene editing technique, Crispr-Cas9, could help create a treatment for muscular dystrophy, according to a new, early study published in the journal Science.Read more

Gene editing propels search for Duchenne muscular dystrophy cure

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion and analysis. After decades of disappointingly slow progress, researchers have taken a substantial step tow...

Manipulating cell signaling for better muscle function in muscular dystrophy

Researchers report on their discovery of a way to bypass faculty cell signalling that leads to muscle damage in Duchene muscular dystrophy. This work suggests a new therapeutic strategy for patients w...

Ottawa researchers discover that Duchenne muscular dystrophy is a stem cell disease

A new study from The Ottawa Hospital and the University of Ottawa is poised to completely change our understanding of Duchenne muscular dystrophy and pave the way for far more effective treatments. Th...

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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...

Respiratory management of patients with Fukuyama congenital muscular dystrophy.

Fukuyama congenital muscular dystrophy (FCMD), characterized by intellectual impairment associated with cortical migration defects, is an autosomal recessive disorder caused by mutation in the fukutin...

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...

A Novel Mutation in DMD (c.10797+5G>A) Causes Becker Muscular Dystrophy Associated with Intellectual Disability.

Severe intellectual disability has been reported in a subgroup of patients with Duchenne muscular dystrophy but is not typically associated with Becker muscular dystrophy.

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...

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