Informationsfilm om hur Spinraza (nusinersen) administreras

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.

A critical review and meta-analysis for more than 2600 SMA patients worldwide. To what extent can a treatment change the natural history outside of the clinical trial profile?

With the growing body of evidence and an evolving treatment landscape, the classification of subtypes needs a revision and Real-World Evidence becomes more and more important.

This short PubExplainer gives you an overview of the results of the March 29, 2019 interim analysis of the NURTURE-study in presymptomatic infants treated with nusinersen by De Vivo et al.

This PubExplainer, on a publication by Hagenacker et al., provides an overview of one of the largest and most diverse real-world studies in adult SMA-patients. The patients were followed at 10 centers in Germany.

This PubExplainer, by Maggi et al., provides an overview of one of the largest real-world studies in adult SMA-patients published so far. The patients were followed at 18 centers in Italy.

In just a few minutes, this PubExplainer gives you an overview of the results of the independent meta-analysis performed by Coratti et al., on real world treatment effect with nusinersen.

Dr. Bartels explains how to distinguish between perceived fatigue and fatigability and how the endurance shuttle tests can be used to assess fatigability across the SMA spectrum.

Dr. Duong explains why a novel outcome measure is needed for adult non-sitters, how it was developed using a human-centered approach and her experience of using the ATEND both in clinic and remotely.

Dr. Rodrigue reviews a Canadian outcome measure toolkit för adults with SMA. He discusses the needs unmet by the existing assessment scales and how digital assessments can be an important complement.

Contains instructions on how to perform CHOP-INTEND and how to complete the test paper

Contains instructions on how to perform HFMSE and how to complete the test paper

Contains instructions on how to perform RULM and how to complete the test paper

This video was developed by Biogen together with other stakeholders and tells the story of Zac, an (imaginary) SMA patient. This video can be used to explain SMA to younger patients.