SMA Newsroom

TreatmentsNov 5, 2026

Information regarding recent Zolgensma safety concerns

SMA Europe is aware of information currently circulating regarding Zolgensma and reports of tumours in children who have previously received treatment.
 
We know that this information may be deeply concerning for families, particularly for parents whose children have already received Zolgensma or who are currently considering treatment. Families need clear, reliable and understandable information and should not be left to piece together a potential safety concern from social media, rumours or incomplete reports.
 
We understand that the relevant regulatory authorities are actively involved and are currently assessing the available evidence and determining what further steps may be needed. We trust in the regulatory processes established to evaluate safety signals and to take the necessary measures to protect patients and ensure the continued safety of medicines on the market.
Nov 3, 2026

Community update: Scholar Rock

Scholar Rock, resubmitted the Marketing Authorisation Application for apitegromab to the EMA. The company anticipates a CHMP opinion in the first half of 2027.

Nov 3, 2026

Community update: argenx

argenx has resubmitted the SPARKLE clinical trial application for authorisation in the EU, with a regulatory decision expected in early 2027. SPARKLE is a Phase 2 study evaluating adimanebart, an investigational MuSK agonist, in children and adolescents with SMA aged 5 to under 18 years.