Extension of natural history study: an important step toward better care and clinical trials

The Prinses Beatrix Spierfonds, Stichting Voor Sara, CureCMD and Giving Strength are continuing their collaboration with Radboudumc by extending and expanding the natural history study on the rare muscle diseases LAMA2-MD and SELENON-RM. Thanks to this joint investment, patients can be followed over a longer period of time, generating crucial knowledge for improved care and the preparation of future clinical trials.

The study, which was initiated several years ago in part at the initiative of Stichting Voor Sara, represents an important building block in the pathway toward therapy development. By carefully mapping the natural course of these muscle diseases, a solid scientific foundation is created that will allow new treatments to be reliably tested in the future. At the same time, the study already delivers direct benefits for patient care, as physicians gain better insight into the symptoms and care needs of children and adults with LAMA2-MD and SELENON-RM.

The Extended LAST STRONG study follows patients over multiple years and systematically documents changes in, among other things, muscle strength, respiratory function and overall functioning. This type of research is essential to determine which outcome measures are most suitable for future therapy trials. In the first phase of the study, dozens of patients participated, yielding valuable insights, for example in the area of cardiac and bone-related complications in this patient population.

“It already helps us improve care and prepares us for clinical trials.”

Thanks to the extended funding, follow-up assessments can now take place three and five years after the start of the study. Patients will be invited again for comprehensive clinical evaluations, including neurological examinations, pulmonary function tests, imaging and functional assessments. These data form the basis for future clinical trials and contribute to making the Netherlands a more attractive location for international drug research, enabling patients to participate in studies closer to home.

Impact on care and the future
An important focus of the project is the integration of the knowledge gained into routine clinical care. By structurally embedding measurement methods and insights into clinical protocols, care for people with LAMA2-MD and SELENON-RM becomes more future-proof and improved (inter)national guidelines can be developed.

Principal investigator Corrie Erasmus emphasizes the importance of this work:

“The aim of this research is to map the natural course of these muscle diseases over several years and to select outcome measures that can later determine whether new therapies are effective. This already helps us improve care and prepares us for clinical trials.”

The collaboration between patient organizations, funding bodies and researchers demonstrates that joint efforts make a real difference. By combining knowledge development with improvements in care and trial readiness, new perspectives are created for patients and their families—today and in the future.