Introduction to SMA research
Intro
SMA Europe promotes and generates patient-relevant data
The development of treatments for SMA involves the transformation of knowledge through successive fields of research, from a basic science discovery, to public health impact, a complex process that requires both research (e.g., bench-work and clinical trials) and non-research activities (e.g., implementation).
Find out more about basic science discovery in SMA and about drug development here for SMA.
Our mission is to be active and progressive in the search for treatments for spinal muscular atrophy (SMA). We do this through promoting and generating patient-relevant data.
Through our research programme, we:
- Seek to set patient-relevant research priorities
- Promote these patient-relevant research priorities in our Call for Research Proposals and our scientific SMA congresses.
- Systematically research and assess the needs and wants of patients
- Identify data gaps that are relevant to patients and fill those by:
- producing our own patient-relevant research projects and publishing the outcome in peer-reviewed journals
- stimulating, (collaboratively) supporting and funding research that addresses these gaps
- facilitating communication between stakeholders in this field.
In focus
Educating our members
We also build our members’ capacity to understand the relevance and processes of research, to allow them to become partners in funding research and in meaningfully contributing to discussions and solutions. In so doing, we make sure SMA research delivers on patients’ unmet needs from a clinical, care and quality of life perspective.
We do this because we believe that developing a treatment that can truly help improve the lives of people living with SMA should be rooted in a firm understanding of the challenges those people face in their daily lives, their needs and the trade-offs they are willing to make to gain relief. To ensure the creation of valuable treatments, all aspects of the health care system, including research prioritisation, product development, trial design, regulatory approval, access, reimbursement and treatment decisions, will need to align with their needs.
The pages in this section refer to patient-relevant biomedical research.
See our other strategic pillars for initiatives in the other disciplines.