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Search Results for "Ullrich Muscular Dystrophy Life Expectancy"

21:43 EDT 28th April 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...

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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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...

Scientists reveal how osteopontin ablation ameliorates muscular dystrophy

Removing an immunomodulatory protein called osteopontin improves the symptoms of mice with muscular dystrophy by changing the type of macrophages acting on damaged muscle tissue, according to a paper ...

Decoding sugar molecules offers new key for combating muscular dystrophy

A group of Japanese scientists have succeeded in decoding a sugar molecule and clarifying a mechanism linked to muscular dystrophy. Their discovery has potential implications for muscular dystrophy tr...

The State of the Muscular Dystrophy Drug Pipeline

The path to marketing an effective drug for Duchenne muscular dystrophy (DMD) hit another snag earlier this week.Read more about The State of the Muscular Dystrophy Drug PipelineComments

UCLA scientists reveal how osteopontin ablation ameliorates muscular dystrophy

(Rockefeller University Press) Removing an immunomodulatory protein called osteopontin improves the symptoms of mice with muscular dystrophy by changing the type of macrophages acting on damaged muscl...

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

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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...

Determinants of the incidence of Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD), an X-linked disorder, is the most common muscular dystrophy with an incidence in boys of about 200 per million births. It presents in early childhood leading to deat...

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