First Chinese conference on translational research in DMD

7 - 9 Apr 2011

Guangzhou, China

Networking Duchenne muscular dystrophy research and care in China

Conference report by Professor Haifang Yin, Tianjin Medical University

Conference banner

After six months of intensive preparation, the first ever Chinese DMD conference was successfully held from 7-9 April 2011in Guangzhou, China, where a group of leading international neuromuscular specialists and DMD patient organizations joined Chinese healthcare professionals, patient groups and researchers to take part in the 1st Chinese Conference on Translational Research in Duchenne Muscular Dystrophy.

Around 300 people from across the country, including clinicians, healthcare professionals, researchers and patients and families, as well as medical students from Sun Yat-sen University and Tianjin Medical University, participated in a two-day scientific programme drawn up jointly by TREAT-NMD (Professor Volker Straub, Newcastle University) and the leaders of the local organising committee (Professor Cheng Zhang, Sun Yat-sen University, Guangzhou and Professor Haifang Yin, Tianjin Medical University). The scientific programme was followed by a dedicated family day led by the United Parent Projects Muscular Dystrophy (UPPMD - Pat Furlong and Elizabeth Vroom) and Chinese representatives.

There are an estimated 40 thousand DMD patients in China – a sixth of the total world Duchenne population – and the burgeoning interest in translational research in the country combined with the availability of international consensus guidelines on care standards gave local organisers the feeling that this was the right time to grasp the opportunity to bring Chinese healthcare professionals, researchers and patients together and engage them in a DMD networking initiative that will help catalyse Chinese research towards treatments for DMD and also give Chinese boys with Duchenne access to better care and to future trials and therapies.

Scientific conference

The conference programme covered the latest updates in research, clinical trials and care in Duchenne muscular dystrophy (DMD) and coincided with the official launch of the published Chinese version of the DMD family guide to the international care standards for the condition, which has been translated into Chinese (both traditional and simplified) by the Hong Kong Paediatric Neurology Association. The printed versions of the guide arrived "hot off the press" in the middle of the conference itself, and families flocked to get their hands on a copy of this eagerly awaited document.

Presentations on outcome measures, patient registries, recent clinical trials and cutting-edge antisense oligonucleotide research, while highlighting the current optimism that therapies for DMD are on the horizon, also focused attention on the many challenges that remain before a treatment that has interesting preclinical results in the lab can actually make it to the stage of being a marketed therapy available to patients.

Chinese speakers revealed the current situation in patient care in China, highlighting its positive aspects, such as the availability of expertise and efforts to keep up with international standards, and challenges including lack of universal availability of genetic diagnosis and the variability of access to care between regions. Presentations from the international patient advocacy organisations involved in UPPMD served to emphasise to the professional audience the importance of equal partnerships between clinicians and patients and their families and showed what parents and families can achieve by working together.

Family day

The family day on the Saturday was a highly interactive event that gave patients and families the opportunity to ask the Chinese and international specialists specific questions about trials, therapies and care. Research and care updates from international speakers were given in language accessible to families, and the US and European Parent Project speakers gave a clear message of patient and family strength and leadership. The Chinese patient groups are now considering setting up their own website and working together to raise the awareness of DMD in China as well as planning a longer dedicated "family conference" in the coming years.

Panel session on Chinese networking

Panel sessionThe scientific programme closed with a panel session led by local organisers Professors Cheng Zhang and Haifang Yin that brought together clinicians from specialist centres across China (including Hong Kong) for a discussion about the creation of a Chinese neuromuscular network.  The aim of such an initiative would be to improve integration into international initiatives in the neuromuscular field, prepare for participation in future clinical trials, and provide an infrastructure for dissemination of information relating to DMD and for the implementation of best-practice care.

The participants concluded that it will be important as a next step to engage more clinicians and the involved medical centres from China and Hong Kong for mutual exchange and sharing of clinical experience on care, treatment and rehabilitation services for DMD children. Academic meetings involving medical and multidisciplinary teams from China, Hong Kong and overseas experts will be helpful to ensure mutual learning, updates on research findings, sharing expertise and enhancement of network collaboration. With the high interest in the translated DMD family guide proving its importance to Chinese families, the Hong Kong Paediatric Neurology Association are now also planning to translate the family guide for SMA in the coming year and distribute it to SMA families across Hong Kong and China. Interest in establishing a Chinese and Hong Kong patient registry for DMD and SMA that feeds into the global TREAT-NMD registries initiative has been evident for some time, and this conference has provided a further incentive to make progress in this regard in the coming months.

The enthusiasm of the panellists and audience members to increase networking was evident and concrete proposals have already been put forward, with a follow-up meeting planned at the end of May during the Chinese Conference of Neurology.

This conference was made possible thanks to the generous support of its sponsors. We would like to take this opportunity to thank our sponsors as well as all the speakers who gave so generously of their time and expertise and brought hope and the real opportunity for future progress to so many DMD families and the medical community in China.

International Speakers:

Annemieke Aartsma-Rus, Netherlands
Doug Biggar, Canada
Kevin Campbell, USA
Cristina Csimma, USA
Michelle Eagle, UK
Richard Finkel, USA
Pat Furlong, USA
Kathryn North, Australia
Markus Rüegg, Switzerland
Thomas Sejersen, Sweden
Volker Straub, UK
Jan Verschuuren, Netherlands
Elizabeth Vroom, Netherlands
Matthew Wood, UK

Local Organizing Committee:

Prof. Cheng Zhang (The First Affiliated Hospital of Sun Yat-sen University, Guangzhou)
Prof. Haifang Yin (Tianjin Medical University, Tianjin)
Dr Shanwei Feng (The First Affiliated Hospital of Sun Yat-sen University, Guangzhou)
Prof. Yun Yuan (The First Affiliated Hospital of Peking University, Beijing)
Prof. Zhuolin Liu (The First Affiliated Hospital of Sun Yat-sen University, Guangzhou)
Prof. Xihua Li (Shanghai Jiao tong University, Shanghai)
Prof. Chuanzhu Yan (Shandong University, Jinan)
Prof. Shiming Jin (Chinese Association of Integrative Medicine)
Dr Juan Yang (The First Affiliated Hospital of Sun Yat-sen University, Guangzhou)
Dr Jiqing Cao (The First Affiliated Hospital of Sun Yat-sen University, Guangzhou)

Hong Kong collaborator:

Dr Sophelia Chan (Neuromuscular working group, Paediatric Neurology Association of Hong Kong)

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