Research News

PTC Therapeutics announce new clinical trial

This page has information on the PTC Therapeutics announce new clinical trial.

Myo-029 is safe but does not increase muscle strength

This page has information on the results of a phase I/II trial to test the safety and tolerability of Myo-029.

New transplant technique to treat mitochondrial myopathy

Funded by the Muscular Dystrophy Campaign, Professor Turnbull and his team at the University of Newcastle are developing a technique which in time might be used to prevent the transmission of mitochondrial myopathies from one generation to the next.

Scientists reverse myotonia in a mouse model of myotonic dystrophy

Researchers from the University of Rochester Medical Center have been able to reverse the symptoms of myotonia in a mouse model for myotonic dystrophy. The results of this study have been published online today in the Journal of Clinical Investigation

New mutations causing congenital myopathies

Professor Francesco Muntoni and his colleagues at Imperial College and the Hammersmith hospital have found nine novel mutations that can cause congenital myopathies. This result was published in the high impact journal Brain earlier this year.

HFEA reaches decision on cybrid embryo research

The Human Fertilisation and Embryology Authority (HFEA) yesterday reached an in principle decision to allow the possible use of “cybrid” embryos for research.

New evidence describing the genetic mechanism causing facioscapulohumeral muscular dystrophy (FSHD)

New evidence for a molecular mechanism causing facioscapulohumeral muscular dystrophy (FSHD)

Anti-hypertensive drug may help slow progress of Duchenne muscular dystrophy

Dr Harry Dietz and his colleagues at Johns Hopkins University School of Medicine have published a paper in the prestigious scientific journal Nature Medicine which shows that a drug called losartan slows the progress of muscle degeneration in mdx mice - a model of Duchenne muscular dystrophy.

Santhera licenses Omigapil for treatment of rare neuromuscular disorders

Santhera Pharmaceuticals, a Swiss based company with special focus on the development of treatment for neuromuscular disorders, announced on Monday a licensing agreement with Novartis to test their compound omigapil for the treatment of congenital muscular dystrophy.

Not all muscle stem cells are equal

A recently published article in Cell, by Michael Rudnicki and his team at the Ottawa Health Research Institute, Canada has provided evidence that not all muscle stem cells – or satellite cells – have the same biological characteristics. Their findings are crucial for understanding satellite cell biology which is important for exploring new avenues for the development of stem cell therapies for neuromuscular disorders.

Steroids prolong walking in Duchenne muscular dystrophy

A recent study, by Dr J Kissel and his colleagues at Ohio State University, has shown that the use of corticosteroids in Duchenne muscular dystrophy can prolong walking on average by three years as well as help prevent scoliosis. It appears that with these benefits, however, comes an increase in the number of bone fractures the boys are likely to suffer from.

Potential new therapeutic target to improve symptoms for Duchenne MD

In a recent article published in the scientific journal Genes and Development, Dr Bruce Spiegelman (Dana-Farber Cancer Institute) and colleagues report on the characterisation of a protein, called PGC-1alpha. This protein appears to play an important role in the biology of muscle function and they suggest it may be a ‘target’ for further research into its potential as a treatment for Duchenne muscular dystrophy.

PTC124 "Daily Pill for Duchenne" - Research summary

Promising results were published this week in the science journal “Nature” about the safety and efficacy of a drug which has the potential to develop into a treatment for 10-15% of boys with Duchenne muscular dystrophy. This article provides an overview of that research and answers some key questions.

Department of Health Funded Project: Progress Report, November 2006

Information about the MDEX Consortium, and an update from the team

Preimplantation Genetic Diagnosis (PGD)

The recent research update published by Muscular Dystrophy Campaign on preimplantation genetic diagnosis

FSH animal model

Deep explanation about how the Facioscapulohumeral muscular dystrophy (FSH) works in humans, and the research of human genes in mice

Department of Health Funded Project: Progress Report, August 2005

Department of Health Funded Project progress report August 2005, last report April 2005

Molecular Patch Therapy Q & A - 07/05

This question and answer document is aimed at explaining the ‘molecular patch’ technique as a therapy for Duchenne muscular dystrophy and the recent awarded funds for this innovative research from the Department of Health (DoH).

Department of Health Funded Project: Progress Report, April 2005

An update on the work of the Department of Health funded research project restoring dystrophin expression in Duchenne muscular dystrophy

Department of Health Funded Project: Progress Report, December 2004

Department of Health Funded Project: “Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A UK Consortium for Preclinical Optimisation and a Phase I/II Clinical Trial Using Antisense Oligonucleotides” Progress Report 1: December 2004

Ventilation in Duchenne muscular dystrophy. Questions & Answers

A paper by Michelle Eagle, Kate Bushby et al has shown that non-invasive nocturnal ventilation can have a significant effect on extending the life span of children with Duchenne muscular dystrophy.

Duchenne muscular dystrophy - 'molecular patches' as a therapy - Application to the Department of He

Q & A on 'molecular patch' research as a therapy for Duchenne muscular dystrophy, and the application to the DoH