This year, International Myology Congress was held for the 7th time, from September 12th to 15th, 2022. Researchers and therapists from all over the world gathered in Nice, France, to advance knowledge of neuromuscular diseases.
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.