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Download PDF version CEO Edwards hugely excited after DMD 'breakthrough' "The significant reduction in muscle damage coupled with the increase in utrophin expression seen in PhaseOut DMD trial patients at 24 weeks is very encouraging as it suggests ezutromid may slow the relentless cycle of muscle fibre degeneration and regeneration that is a hallmark of DMD," said Professor Francesco Muntoni.
The headlines from the study Developmental myosin is a biomarker of muscle damage and is found in repairing fibres. Duchenne Muscular Dystrophy is a rare muscle-wasting disease that affects boys.
Dystrophin replacement The protein is functionally and structurally similar to dystrophin, which is lacking in children with the disease.
Preclinical studies revealed the continued expression of utrophin had a meaningful, positive effect on muscle performance. The current trial appears to bear out the current findings. The surprise is that its effects of ezutromid have been detected so early in the process.
Investors will be updated next on the trial in the third-quarter, when PhaseOut hits the week stage.The RARE List™ is comprised of approximately 7, different rare diseases and disorders affecting more than million people worldwide. More RARE Facts™ can be found here.
We are often asked why some common diseases are included on the RARE List™. The answer is two fold:This is a list of rare diseases defined in the United States where a prevalence of less than , cases is the.
Specific Genetic Disorders. Many human diseases have a genetic component. Some of these conditions are under investigation by researchers at or associated with the National Human Genome Research Institute (NHGRI).
CPHI/CEAR CTSA Pilot Grant Program. Application due date: Friday, May 6, at pm The Center for Public Health Initiatives (CPHI) and the Community Engagement and Research (CEAR) core of the Clinical and Translational Science Award (CTSA) at the University of Pennsylvania are collaborating on a pilot grant program to fund research with public and community health relevance.
HACKENSACK, N.J., Dec. 7, /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), along with collaborators the FSH Society and the Foundation to Eradicate Duchenne (FED), are.
Action Duchenne is the UK's first charity dedicated solely to improving the lives of all affected by Duchenne muscular dystrophy (DMD).
PTC Therapeutics Receives Formal Dispute Resolution Request Decision from the FDA's Office of New Drugs.