Understanding European patient expectations towards current therapeutic development in spinal muscular atrophy


Following the 2017 approval of a first SMA treatment by the European Medicines Agency, SMA Europe launched a Europe-wide survey with the goal of understanding patients’ treatment expectations, realities of daily living and access to clinical trials and therapy and how this varied according to parameters such as age and disease severity.

A response rate of 31% yielded 1474 completed surveys from 26 European countries. In line with findings from a 2015 SMA Europe-led survey, participants considered stabilisation of their condition to be progress. Notably, responses indicated that the current classification of SMA at diagnosis by ‘type’, often does not reflect current mobility level. Large gaps in treatment access were identified that varied in particular between age and disease severity groups, yet there was high interest in clinical trial participation. In addition, alternative treatment options, including combination therapies, are now expectations.

These perspectives should be central considerations through the research and development processes of new SMA therapies, through data generation and discussions on access to therapies. Results from this survey indicate that collaboration between stakeholders is essential to the foundation upon which innovative approaches for SMA treatments and access can be explored.

Authors: Nicole Gusset, Caroline Stalens, Eva Stumpe, Lori Klouvi, Alexandre Méjat, Marie-Christine Ouillade and Mencía de Lemus

Link: Understanding European patient expectations towards current therapeutic development in spinal muscular atrophy

A Decision for Life – Treatment decisions in newly diagnosed families with spinal muscular atrophy (SMA)


European neuromuscular experts presented eleven consensus statements covering qualification, patient selection, safety considerations and long-term monitoring, in order to aid the rational use of Zolgensma™ for the treatment of SMA (Kirschner, et al., 2020). In response to this consensus statement, a group of patient representatives from SMA Europe highlight considerations that must be taken into account when considering treatment.

Authors: N. Gusset, Y. Erbas, O. Germanenko, K. Rucinski, E. Stumpe and M. de Lemus.

Journal: Neuromuscular Disorders

Link: 10.1016/j.ejpn.2020.11.003

New treatments for spinal muscular atrophy open-up new challenges for European & national institutions


SMA Europe discusses how new treatments for spinal muscular atrophy open up new challenges for European and national institutions

Authors: Marie-Christine Ouillade & Alexandre Méjat.


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Disease impact on general well-being and therapeutic expectations of European Type II and Type III spinal muscular atrophy patients


Authors: F. Rouault, V. Christie-Brown, R. Broekgaarden, N. Gusset, D. Henderson, P. Marczuk, I. Schwersenz, G. Bellis, C. Cottet .

Journal: Neuromuscular Disorders
DOI: 10.1016/j.nmd.2017.01.018