Search Results for "Ullrich Muscular Dystrophy Life Expectancy"

12:26 EST 28th February 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...

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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Muscular Dystrophy Partnering

The Muscular Dystrophy Partnering report provides understanding and access to the muscular dystrophy partnering deals and agreements entered into by the worlds leading healthcare companies. Trends in ...

Cardiac stem cell therapy may heal heart damage caused by Duchenne muscular dystrophy

(Cedars-Sinai Medical Center) Researchers at the Cedars-Sinai Heart Institute have found that injections of cardiac stem cells might help reverse heart damage caused by Duchenne muscular dystrophy, po...

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 ...

Idera, Parent Project Muscular Dystrophy partner to advance new treatment approach for duchenne muscular dystrophy

Idera Pharmaceuticals and Parent Project Muscular Dystrophy have announced a collaboration to advance Idera's proprietary Toll-like receptor (TLR) technology for the treatment of Duchenne muscular dys...

Idera Pharmaceuticals, Parent Project Muscular Dystrophy collaborate

Idera Pharmaceuticals, a Cambridge, Mass.-based clinical-stage biopharmaceutical company, and nonprofit organization Parent Project Muscular Dystrophy (PPMD), based in Hackensack, N.J., are collabora...

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 ...

Are my muscular dystrophy drugs working?

People with muscular dystrophy could one day assess the effectiveness of their medication with the help of a smartphone-linked device, a new study in mice suggests. The study used a new method to proc...

Idera Pharmaceuticals and Parent Project Muscular Dystrophy Announce Collaboration to Advance New Treatment Approach for Duchenne Muscular Dystrophy

Toll-like receptor antagonism designed to treat muscle inflammation central to disease progression New approach offers potential to treat broad population of patients regardless of their gen...

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Ullrich Congenital Muscular Dystrophy Possibly Related With COL6A1 p.Gly302Arg Variant.

Ullrich congenital muscular dystrophy (UCMD) is characterized by congenital weakness, proximal joint contractures, and hyperlaxity of distal joints. UCMD is basically due to a defect in extra cellular...

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

Blunted cardiac beta-adrenergic response as an early indication of cardiac dysfunction in Duchenne muscular dystrophy.

To determine whether altered beta-adrenergic responses contribute to early cardiac dysfunction in mdx (X-linked muscular dystrophy) mice, an animal model for human Duchenne muscular dystrophy (DMD).

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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