Advertisement

Topics

Search Results for "Ullrich Muscular Dystrophy Life Expectancy"

12:49 EDT 18th October 2017 | BioPortfolio

Matching Channels

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 ...

Matching News

Shortened telomeres linked to dysfunction in Duchenne muscular dystrophy, researchers find

A discovery about muscular dystrophy disorders has been made that suggests new possibilities for treatment. Researchers found that stem cells in the muscles of muscular dystrophy patients may, at an e...

Researchers find shortened telomeres linked to dysfunction in Duchenne muscular dystrophy

Researchers from the Perelman School of Medicine at the University of Pennsylvania have made a discovery about muscular dystrophy disorders that suggest new possibilities for treatment. In a study pub...

Study: Drug Candidate Demonstrates Efficacy in Duchenne Muscular Dystrophy

NewsBiophytis clinical stage drug-candidate sarconeos demonstrates efficacy in preclinical models of Duchenne muscular dystrophy. Duchenne muscular dystrophy is a new 'Orphan' indication for Sarconeos...

Researchers repurpose former experimental cancer therapy to treat muscular dystrophy

Researchers at the National Institutes of Health's National Center for Advancing Translational Sciences (NCATS) and the University of Nevada, Reno School of Medicine (UNR Med) have demonstrated that a...

Duchenne Muscular Dystrophy: The Race For The $1bn Opportunity

With three products now approved in Europe and/or the US for Duchenne Muscular Dystrophy, an FDA decision pending on PCT...   

Researchers replicate FSH muscular dystrophy in mice

A new study published in the journal Nature Communications describes a breakthrough in research related to facioscapulohumeral muscular dystrophy (FSHD). The debilitating genetic disease - which has n...

STAT Plus: FDA panel votes no on PTC’s Duchenne muscular dystrophy drug

An FDA advisory panel is reviewing a controversial therapy for Duchenne muscular dystrophy from PTC Therapeutics (PTCT).

University of Minnesota researchers replicate FSH muscular dystrophy in mice

(University of Minnesota) A new study published in the journal Nature Communications describes a breakthrough in research related to facioscapulohumeral muscular dystrophy (FSHD). The debilitating gen...

Matching PubMed Articles

Translation and validation of the Life Satisfaction Index for Adolescents scale with neuromuscular disorders: LSI-A Brazil.

To validate the Life Satisfaction Index for Adolescents (LSI-A) scale, parent version and patient version, for Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA) and limb-girdle muscular...

Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy.

Dominant-negative mutations in the genes that encode the three major α chains of collagen type VI, COL6A1, COL6A2, and COL6A3, account for more than 50% of Ullrich congenital muscular dystrophy patie...

Parents' Reactions to the Diagnosis of Duchenne Muscular Dystrophy: Associations Between Resolution, Family Functioning, and Child Behavior Problems.

Duchenne muscular dystrophy (DMD) is the most frequent inherited form of muscular dystrophy during childhood. DMD is a severe and progressive disease. Children initially have no symptoms, but the diag...

The relationship between quality of life and health-related quality of life in young males with Duchenne muscular dystrophy.

This study investigated the relationship between quality of life (QoL) and health-related quality of life (HRQoL) and assessed factors other than health that contribute to differences in QoL in young ...

Upper limb functional assessment scale for children with Duchenne muscular dystrophy and Spinal muscular atrophy.

Duchenne muscular dystrophy (DMD) and Spinal muscular atrophy (SMA) causes significant disability and progressive functional impairment. Readily available instruments that assess functionality, especi...

Search Whole site using Google

Loading
Quick Search
Advertisement
 
Advertisement Advertisement