World Muscle Society Congress
Many thanks to Nigel Laing (Western Australia), Rebecca Fairclough (Oxford) and Debbie Hicks (Newcastle) who helped with the preparation of this report.
The World Muscle Society is an international society of scientists and clinicians for the promotion and dissemination of knowledge in the neuromuscular field. It was founded in 1995 and holds a yearly meeting to facilitate the exchange of information. The 13th Congress of the World Muscle Society continued the trend of expansion of this meeting in recent years. More than 550 muscle disease researchers attended the meeting in Newcastle-upon-Tyne, at the excellent Sage Centre and Hilton Hotel, Gateshead.
Below we give an overview of the meeting and some of the highlights.
Contents:
- New insights into facioscapulohumeral muscular dystrophy and myotonic dystrophy
- Advances in the understanding and treatment of myasthenic disorders
- Therapeutic advances in neuromuscular disorders
- Late breaking news and conclusions
New insights into facioscapulohumeral muscular dystrophy and myotonic dystrophy
The first day included excellent update talks on facioscapulohumeral muscular dystrophy (FSH). Three talks described the emerging theories on how the DNA mutation causes the symptoms of FSH. This reflects the state of FSH research at the moment and hopefully all of these different approaches will find a way forward to understand this complex condition. Also, in the late breaking news session on the final day of the conference a new technological advancement in the diagnosis of FSH was presented.
Later, Charles Thornton suggested that effective treatment for myotonic dystrophy may, with luck, be closer than we might have thought. He suggested that morpholinos, similar to those used in exon skipping, could be used.
Advances in the understanding and treatment of myasthenic disorders
On the second day myasthenic syndromes were covered by the world’s leading experts in these diseases including Andrew Engel (USA). Muscular Dystrophy Campaign grantee Professor David Beeson also talked about congenital myasthenia - the difficulties in diagnosis and some of the gene mutations involved.
Therapeutic advances in neuromuscular disorders
Standardisation of pre-clinical testing of drugs for muscle disease was discussed, an important initiative to allow comparison of different international pre-clinical trials, and ensure the quality of pre-clinical research.
Recent advances in the design of exon-skipping drugs and the challenges being faced were presented by Steve Wilton. Luis Garcia from France gave a talk on trans-splicing technology. This is a new technology in the early stages of development which rather than skipping a mutation it replaces it with the correct DNA sequence.
The preclinical results of two drugs - Idebenone (for Duchenne muscular dystrophy) and Omigapil (for congenital muscular dystrophy) in mouse models were presented. The pre-clinical testing seemed promising with improvements in symptoms in the mice.
Late breaking news and conclusions
The “late breaking news” session presented new gene mutations causing limb girdle muscular dystrophy 2L (LGMD2L), myofibrillar myopathy, X-linked myopathy with excessive autophagy (XMEA) and X-linked Emery-Dreifuss muscular dystrophy.
A major platform for communication at this conference is the poster sessions. There were multiple parallel poster session presentations at multiple times during the meeting, with hundreds of posters covering an ever-expanding range of muscle disease topics. At least 16 of the posters presented work funded by the Muscular Dystrophy Campaign.
The Congress closed with the awarding of the multiple prizes available to young presenters. One of which was Muscular Dystrophy Campaign grantee Debbie Hicks from Newcastle who won one of the awards for best oral presentations.
Overall, the impression is of enormous energy in the field of skeletal muscle disease research, with considerable optimism about future successful therapies. Details of this and future WMS Congresses may be obtained from the World Muscle Society.
