Following the success of the first masterclass in 2015, this year’s two-day expert masterclass was held in Warsaw, Poland on the 8-9th September. The masterclass provided a high-quality scientific and educational meeting for paediatric neurologists and people involved in diagnosing and/or managing the care of patients with DMD.
The masterclass attracted 50 delegates from across 27 countries. The masterclass started with a presentation from Filippo Bucella (Duchenne Parent Project Onlus, Italy) who shared the experiences of families with DMD. Additional presentations were given by Academic experts, including a review of the current understanding of the natural history of DMD and best practice for the management of patients at various stages of ambulation and non-ambulation. Experts also provided updates on therapeutic advances in DMD and potential implications for clinical practice.
The masterclass format further included interactive workshops, which provided the opportunity for attendees to discuss methodologies for the evaluation of treatment outcomes, as well as discuss the regional differences and challenges in the diagnosis and management of DMD patients.
Finally, Elizabeth Vroom (Duchenne Parent Project Netherlands and United Parent Project Muscular Dystrophy) provided an overview of the support patient advocacy groups provide families of DMD patients, as well as the important role they play in helping DMD patients gain better access to DMD treatment.
TREAT-NMD received an unrestricted educational grant from PTC Therapeutics International Ltd for the organisation of this meeting.