Search Results for "Duchenne Muscular Dystrophy Treatment"
Original Source: Daiichi Sankyo opens office of Orphan Disease Treatment Institute
Daiichi Sankyo Company has opened the Orphan Disease Treatment Institute office inside the Daiichi Sankyo Shinagawa R&D center.
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Muscular dystrophy is a group of degenerative inherited disorders causing muscle weakness and loss of muscle tissue. The different types are Becker muscular dystrophy, Duchenne muscular dystrophy, E...
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...
Antisense therapy is a form of treatment for genetic disorders or infections. When the genetic sequence of a particular gene is known to be causative of a particular disease, it is possible to synth...
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Parent Project Muscular Dystrophy funds Duchenne muscular dystrophy study
Parent Project Muscular Dystrophy (PPMD) has granted $175,000 in supplemental funds to advance the Duchenne muscular dystrophy study conducted by Dr. Craig McDonald of the University of California, Da...
Muscular Dystrophy Drug Helps Boys Walk Further
An experimental drug, eteplirsen, helped boys with Duchenne Muscular Dystrophy walk considerably better half way through a clinical trial, Sarepta Therapeutics Inc. announced today. Duchenne Muscular...
Potential New Treatment For Duchenne Muscular Dystrophy
Scientists at UCLA have identified a new compound that could treat certain types of genetic disorders in muscles. It is a big first step in what they hope will lead to human clinical trials for Duchen...
Muscular Dystrophy Therapies Reviewed
Leading muscular dystrophy researcher Dean Burkin, of the University of Nevada School of Medicine summarizes the impact of a new protein therapeutic, MG53, for the treatment of Duchenne muscular dystr...
Researchers review muscular dystrophy therapies
Leading muscular dystrophy researcher Dean Burkin, of the University of Nevada School of Medicine summarizes the impact of a new protein therapeutic, MG53, for the treatment of Duchenne muscular dystr...
Laminin-111 protein therapy could treat Duchenne muscular dystrophy
A novel treatment in development at the University of Nevada School of Medicine for the most common form of muscular dystrophy is advancing towards human trials with a $308,000 boost from the Muscular...
Pioneering research into Duchenne muscular dystrophy secures commercial funding
(Royal Holloway, University of London) Royal Holloway University is giving money generated by its research into Duchenne muscular dystrophy to the Muscular Dystrophy Campaign. Families affected by the...
Dantrolene may help combat Duchenne muscular dystrophy in boys
Drugs are currently being tested that show promise in treating patients with Duchenne muscular dystrophy (DMD), an inherited disease that affects about one in 3,600 boys and results in muscle degenera...
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Can outcomes in Duchenne muscular dystrophy be improved by public reporting of data?
To review current approaches for obtaining patient data in Duchenne muscular dystrophy (DMD) and consider how monitoring and comparing outcome measures across DMD clinics could facilitate standardized...
Importance of SPP1 genotype as a covariate in clinical trials in Duchenne muscular dystrophy.
To test the effect of the single nucleotide polymorphism -66 T>G (rs28357094) in the osteopontin gene (SPP1) on functional measures over 12 months in Duchenne muscular dystrophy (DMD).
Review of Duchenne Muscular Dystrophy (DMD) for the Pediatricians in the Community.
Duchenne muscular dystrophy (DMD) is a life-limiting neuromuscular disease of young boys. Pediatricians in the community may be involved in care of children affected by DMD and faced with challenging...
Functional changes in Duchenne muscular dystrophy: A 12-month longitudinal cohort study.
The aim of the study was to assess different outcome measures in a cohort of ambulant boys with Duchenne muscular dystrophy (DMD) over 12 months in order to establish the spectrum of possible changes...
