FundAME promotes an international workshop on contractures at the International Scientific Congress on SMA

FundAME will host the workshop “Contractures in SMA: From Scientific Insights to Therapeutic Targets” during the International Scientific Congress on SMA organized by SMA Europe.

The session will be chaired by Tom Crawford, Professor of Neurology at Johns Hopkins University School of Medicine, an internationally recognized leader in neuromuscular neurology.

The workshop will feature an outstanding international panel of speakers:

• Tina Duong, Director of Clinical Outcomes Research & Development, Neurology – Stanford University

• Jordi Diaz-Manera, Professor and researcher – John Walton Muscular Dystrophy Research Centre, Newcastle University

• Olga Tapia, Professor and Principal Investigator – Universidad de La Laguna

• Ying Qian, Director – SMA Foundation

• Mencía de Lemus, CEO – FundAME

The workshop will bring together leading international experts in clinical and basic research, alongside patient representatives, to advance knowledge and therapeutic strategies addressing contractures in spinal muscular atrophy (SMA).

A common and disabling complication with no definitive treatment

Contractures are a common and disabling musculoskeletal complication in SMA, reducing functional capacity, causing pain, and significantly impacting the quality of life of individuals living with the condition.

According to data from RegístrAME — the Spanish Patient Registry of FundAME — contractures affect 44% of individuals with SMA in Spain. Their impact is substantial: they limit mobility, increase pain, reduce independence, and add to the daily caregiving burden. Available evidence shows high prevalence particularly in knees, hips, neck, and upper limbs, with relevant functional consequences.

Disease-modifying therapies (DMTs) have transformed the clinical course of SMA. However, contracture management remains a largely unmet clinical need and continues to rely on standards of care developed in the pre-DMT era.

Despite their significant clinical and functional impact, there are currently no pharmacological or non-pharmacological interventions capable of permanently preventing or reversing contractures.

A 360° strategy to transform Contractures in SMA approach

This session is part of the strategic line launched by FundAME in 2025, when the SMA community identified contractures as a priority unmet need.

In response to this gap, FundAME has promoted a comprehensive 360° strategy to transform the approach, built on three complementary pillars:

• Generation of scientific evidence based on data from the National SMA Patient Registry (RegístrAME).

• Promotion of a clinical consensus to update and harmonize current management practices.

• Mobilization of international scientific interest around this critical issue.unmet needs in SMA

Through this workshop, FundAME reinforces its commitment to driving meaningful change and advancing solutions that improve the quality of life of people living with SMA.