What real-world evidence shows on nusinersen in a wide severity spectrum of SMA patients in long-term observation (up to 30 months), including first-ever evidence for efficacy in adults with Type 1 SMA.

How nusinersen treatment can be beneficial over time for children and teenagers with SMA regardless of their age, functional status, SMA type, or the number of SMN2 copies.

Dr. Giorgia Coratti, Neuropsychomotricist, Department of Pediatric Neurology, Catholic University of Sacred Heart, Rome, Italy, is presenting the meta-analysis “Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis”, March 2023. 

Eugenio Mercuri, Professor of Paediatric Neurology and Head of the Paediatric Neurology and Psychiatry Unit of the Gemelli Hospital Catholic University Foundation, Rome, Italy, is talking about the the importance of RWE in SMA (rare diseases) and the importance of registries. 

Dr. Giorgia Coratti, Neuropsychomotricist, Department of Pediatric Neurology, Catholic University of Sacred Heart, Rome, Italy, is presenting the meta-analysis “Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis”.

The aim of this paper was to critically review the literature reporting real world data on motor function in type 2 and 3 patients treated with nusinersen, subdividing the results according to SMA type, age and type of assessment and performing a meta-analysis of the available results.

The aim of this paper was to retrospectively investigate the safety and efficacy of nusinersen in a large cohort of adult Italian patients with spinal muscular atrophy (SMA).

The pubexplainer is a short animated video made by Biogen which summarizes the key findings and conclusions in Hagenacker et al. 2020 (Lancet Neurology). This study is the largest efficacy and safety study of nusinersen in the most diverse SMA adult population studied to date.

This video shows an expert interview with Dr. Tim Hagenacker, Chief Senior Physician of the Clinic for Neurology, Essen University Hospital.  

This video shows an expert interview with Prof. Christoph Kleinschnitz, Director of the Clinic for Neurology, Essen University Hospital. In this video Prof. Kleinschnitz talks about what conclusions doctors involved in SMA diagnostics should draw from the study results.

This presentation was hosted by Anna-Karin Kroksmark during 2020's Listen & Learn meeting. It was a break-out session intended for physiotherapists on habilitation and rehabilitation in SMA.

This presentation was hosted by Anna-Karin Kroksmark during 2020's Listen & Learn meeting. It was a the opening session of 2020's Listen & Learn meeting and was about outcome measures.

This presentation was done by Mar Tulinius and Christopher Lindberg during 2020's Listen & Learn meeting. They shared their perspectives on the paediatric-adult transition process in SMA from their respective centres in Gothenburg.

Bart Bartels is the head of the Child Development and Exercise Center and a senior researcher at the University Medical Centre (UMC) Utrecht in the Netherlands. In 2020 he completed his PhD on the validation of a set of novel fatigability tests in children and adults with SMA. In this Smart Talk, he will discuss how these tests are constructed and what the results can tell us about fatigability in SMA.

CHOP-INTEND - Instructions on how to perform the CHOP-INTEND

HFMSE - Instructions on how to perform the HFMSE

RULM - Instructions on how to perform the RULM