The 13th International Conference was held in New York City between May 15th and 17th, 2017. It was held in the financial district of NYC, near the historic and tragic events of 9/11. This highly stimulating conference takes places every five years and attracts clinicians, basic scientists and researchers from all over the world to discuss their work on myasthenia. This meeting traditionally includes the presentation of results from clinical research, clinical trials and laboratory-based work on myasthenia.

The conference started off with a session on the neuromuscular junction (the sophisticated area between the nerve and the muscle, which we refer to as NMJ). This was chaired by Professor Slater. In myasthenia gravis (MG), it is well-recognised that antibodies to the nicotinic acetylcholine receptor cause havoc to the muscle surface, resulting in the familiar myasthenic weakness. The speakers showed how even in healthy NMJs, the relationship between nerve and muscle is highly dynamic. Many molecules exist in this region, including signalling molecules that communicate the state of affairs between the nerve and the muscle. The state of the acetylcholine receptors is also communicated to the nerve terminal. The NMJ is in a constant state of change, adapting to the environment it is exposed to. A family of proteins called Homer (yes, Homer!) ensure that communication is intact and that the situation adapts accordingly. As we grow older there are changes at the NMJ. The nerve terminal branches out more and the muscle surface region tends to fragment. However, experiments showed that despite this, the NMJ remains efficient.

A session was dedicated to congenital myasthenic syndromes (CMS) - these are distinct from MG. Children are born with muscle weakness and often have difficulties with breathing and feeding. The problem results from a spelling mistake in an inherited gene. We learnt about congenital myasthenic syndromes presenting with limb girdle weakness. This means that patients experience weakness in their arms and legs but not in their face or eyes. It is important to recognise these conditions, since there are different medications that can help patients to become stronger. They are important to distinguish from limb girdle muscular dystrophy - a different condition altogether.

Professor Angela Vincent chaired a session on antibody classifications. We know more about MuSK antibodies since they were identified almost 16 years ago. Professor Evoli discussed how in MuSK-MG, starting treatment early is important for these patients. In some, where the disease is aggressive from the start, the more powerful drug Rituximab is indicated and works very well. We heard about newer antibodies that can be present in myasthenia including antibodies to LRP-4, cortacin and agrin (all molecules that are present at the NMJ). The antibody repertoire is steadily increasing. At present, these antibodies can only be tested on a research basis, but in the future they may become commercially available, making it easier to diagnose "seronegative" patients.

In the clinical trials session, Professor James Howard presented data from the PHASE 3 REGAIN study. In this study, people with myasthenia who are classed as refractory (which means they had not responded to conventional treatments) were treated with the drug eculizumab. Patients rapidly improved from week 2, and sustained improvement until week 26, which was the study period. Eculizumab is a promising drug for MG. The big question is which patients are most likely to benefit from this drug. BeatMG is a multicentre phase 2 placebo-controlled study, now closed to enrolment but study visits are ongoing. The study aims to see whether rituximab is safe to be prescribed in MG and whether it allows significant reduction in steroid dosed after rituximab has been administered.

Professor Gill Wolfe presented data from the recent international thymectomy trial where 126 patients were recruited. Patients who had a thymectomy (where the thymus is removed) required lower doses of steroids and less patients required azathioprine. In this trial, they included patients up to the age of 65. Until recently, we referred patients for thymectomy if they were younger than 40-50 (unless they had a tumour of the thymus gland called thymoma). More data analysis from this trial is underway. Data from thymus tissue obtained from patients in this trial was presented: Forty-six thymus specimens have been obtained. Work is still in progress to determine if the changes in the thymus are dependent on previous exposure to steroid treatment and whether they correlate with the patient's clinical outcome (how well or not they do).

A session was dedicated to treatment guidelines for MG across the world including North and South America, UK and Japan. It was fascinating listening to the different approaches, although ultimately our goals are all identical - to control myasthenic symptoms and induce remission.

This meeting once again met everyone's high expectations. It was an opportunity to meet "old" friends and network. It fuelled and recharged everyone's enthusiasm to continue to work in the field and look after patients with myasthenia across all ages.

Dr Maria Farrugia
Consultant Neurologist
Queen Elizabeth Hospital, Glasgow