Mencía de Lemus, our Spanish delegate, has been appointed as a member of the Committee for Advanced Therapies, representing patients' organisations.

SMA Europe wants to give You a louder voice in research!

We are launching a project called the “SMA Priority Setting Partnership”, in collaboration with the James Lind Alliance, to identify the top 10 unanswered research questions that are a priority to people living with SMA, their carers and healthcare professionals.

We will soon launch a survey which will allow you to tell us what research would matter to you most. Keep a look out!

The ASCEND study is a global clinical trial initiated by Biogen to evaluate if patients who have previously received treatment with risdiplam, an approved SMA drug also known as Evrysdi® (risdiplam), and may not have achieved optimal clinical outcomes (e.g., improvement or delay in disease progression), may benefit from receiving a higher dose of nusinersen, also known as Spinraza® (nusinersen).

The ECRD is recognised globally as the largest, patient-led rare disease policy event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape goal-driven rare disease policies and allow for important and innovative discussions on a national and an international level to take place.