Brazilian/International Conference on Neuromuscular Diseases:
Care and Clinical Trials

17 - 19 Jun 2010

Sao Paulo, Brazil

Networking neuromuscular research and care in Brazil: conference report

From 17-19 June 2010 a group of international neuromuscular specialists joined Brazilian healthcare professionals and patient groups at the Hospital Sirio-Libanes in Sao Paulo, Brazil, to take part in the International Conference on Neuromuscular Diseases: Care and Clinical Trials. Around 350 people from across the country, including clinicians, physiotherapists, orthotics providers, and patients and families, as well as medical students from the Sao Paulo universities, participated in a two-day scientific programme followed by a dedicated family day with research updates for patients and information on physiotherapy and care.

The conference programme covered the latest updates in research and care in Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), including the published international care standards for these conditions. Presentations on outcome measures, patient registries, recent clinical trials and the TREAT-NMD Advisory Committee for Therapeutics, while highlighting the current optimism that therapies for neuromuscular diseases 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. In a field where there can sometimes be much hype over unproven therapies, the realism presented at the conference was greatly valued by the attendees.

Brazilian speakers revealed the current situation in patient care in Brazil, highlighting its positive aspects, such as the availability of expertise and treatment in line with international standards, as well as its 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 UPPMD and SMA Europe served to emphasise the importance of equal partnerships between clinicians and patient groups and showed what parents and families can achieve by working together, while the family day itself was a highly interactive event that gave patients and families the opportunity to ask the Brazilian and international specialists specific questions about trials, therapies and care. The patient groups are now considering planning a longer dedicated “family conference” in the coming years.

The scientific programme closed with a panel session led by local organiser Professor Arnaldo Godoy that brought together government representatives with clinicians from specialist centres across Brazil and Argentina for a discussion about the creation of a Brazilian or indeed a South American 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 NMDs and for the implementation of best-practice care. The enthusiasm of the panellists and audience members was evident and concrete proposals have already been put forward, with a follow-up meeting planned in August during the Brazilian Congress of Neurology.

For further information on the conference itself or future plans in Brazil, please contact Professor Arnaldo Godoy.