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Search Results for "Ullrich Muscular Dystrophy Life Expectancy"

00:42 EST 6th December 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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Casey’s General Store Raises More Than $1.5 Million to Help Send Kids with Muscular Dystrophy to MDA Summer Camp

Chicago, (PRWEB) November 17, 2016 Casey’s General Store locations and customers in its 14-state area rallied together to raise...

Vitamin Could Help Treat Duchenne Muscular Dystrophy

NewsDuchenne is the most common and severe form of muscular dystrophy. Contributed Author: 

CRISPR may hold key to curing muscular dystrophy

At 24, Benjamin Dupree has outlived many people with Duchenne muscular dystrophy…Doctors say the disease is terminal, but they tell you less about living with it…[I]n college, d

FDA greenlights Sarepta Therapeutics’ milestone treatment for Duchenne muscular dystrophy

Exondys 51 doesn’t cure Duchenne muscular dystrophy and will only help a minority of patients.

Medical News Today: Muscular dystrophy: Cancer drug may boost muscle strength for some patients

Researchers reveal how the cancer drug sunitinib may reduce muscle weakness for patients with facioscapulohumeral dystrophy - a form of muscular dystrophy.

FDA tentatively approves first drug for muscular dystrophy

Federal health regulators have granted tentative approval to a highly contested drug for muscular dystrophy that has become a flashpoint in the debate over patient access to experimental medicine.

FDA Oks 1st muscular dystrophy drug; awaits proof it works

Federal regulators on Monday granted tentative approval to the first drug for muscular dystrophy, following an intense public campaign from patients and doctors who pushed for the largely unproven med...

FDA Oks first muscular dystrophy drug; awaits proof it works

Federal regulators on Monday granted tentative approval to the first drug for muscular dystrophy, following an intense public campaign from patients and doctors who pushed for the largely unproven med...

Matching PubMed Articles

Quality of life of patients with Duchenne muscular dystrophy: from adolescence to young men.

This study investigated quality of life (QOL) in adolescent and young men with Duchenne muscular dystrophy (DMD).

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.

Gastrointestinal Dysfunction in Patients with Duchenne Muscular Dystrophy.

In adult patients with Duchenne muscular dystrophy (DMD) life-threatening constipation has been reported. Since gastrointestinal function in DMD has not been rigorously studied we investigated objecti...

Trends in Working Life Expectancy in Europe.

The aim of the article is to analyze past and present developments of working life expectancy (WLE) at age 50 by age, sex, and education in Europe. WLE is also compared with life expectancy (LE) and h...

Diagnosis and aetiology of congenital muscular dystrophy: we are halfway there.

To evaluate the diagnostic outcomes in a large cohort of congenital muscular dystrophy (CMD) patients using traditional and Next Generation Sequencing (NGS) technologies.

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