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Expert interviews from WMS 2022

Expert interviews from WMS 2022

Sharon Aharoni

20-10-2022 | Neurology | Video

microRNAs linked to nusinersen response in SMA

Sharon Aharoni talks us through the results of a study showing that lower baseline levels of two muscle microRNAs measured in the cerebrospinal fluid are associated with better clinical response to nusinersen treatment in patients with type 2 or 3 spinal muscular atrophy.

Funding for independent interviews at WMS 2022 was provided by F. Hoffmann-La Roche Ltd, Basel, Switzerland.

Tina Duong

18-10-2022 | Neurology | Video

Wheelchair-based motor function assessment feasible in SMA

Tina Duong describes the development and initial testing of ATEND, a wheelchair-based assessment of motor function for non-ambulatory individuals with spinal muscular atrophy.

Funding for independent interviews at WMS 2022 was provided by F. Hoffmann-La Roche Ltd, Basel, Switzerland.

Peter Karachunski

18-10-2022 | Neurology | Video

Favorable treatment outcomes in patients with SMA diagnosed through newborn screening

Peter Karachunski reports the findings from a real-world study showing that disease-modifying therapies have favorable safety and effectiveness profiles in infants with spinal muscular atrophy identified through newborn screening.

Funding for independent interviews at WMS 2022 was provided by F. Hoffmann-La Roche Ltd, Basel, Switzerland.

Evelin Milev

18-10-2022 | Neurology | Video

Nusinersen linked to improvements in upper limb strength, function

Evelin Milev talks about an observational study demonstrating improvements in upper limb function and muscle strength among children with type 2 or 3 spinal muscular atrophy treated with nusinersen.

Funding for independent interviews at WMS 2022 was provided by F. Hoffmann-La Roche Ltd, Basel, Switzerland.

Laurane Mackels

18-10-2022 | Neurology | Video

Myostatin levels vary according to SMA patient characteristics

Laurane Mackels discusses the results of a study showing that pretreatment levels of myostatin may be associated with disease type and motor function, and inversely correlated with age and weight, among people with spinal muscular atrophy.

Funding for independent interviews at WMS 2022 was provided by F. Hoffmann-La Roche Ltd, Basel, Switzerland.

Andrea Oliveira-Carneiro

18-10-2022 | Neurology | Video

Altered patterns of functional connectivity identified in SMA

Andrea Oliveira-Carneiro outlines a brain imaging study that found patterns of increased and decreased functional connectivity in patients with spinal muscular atrophy relative to healthy controls, which could represent potential therapeutic targets.

Funding for independent interviews at WMS 2022 was provided by F. Hoffmann-La Roche Ltd, Basel, Switzerland.

Susana Quijano-Roy

18-10-2022 | Neurology | Video

Promising real-world risdiplam outcomes in children with SMA

Susana Quijano-Roy shares her real-world experience of risdiplam treatment in children with symptomatic spinal muscular atrophy, finding favorable short-term outcomes in most patients.

Funding for independent interviews at WMS 2022 was provided by F. Hoffmann-La Roche Ltd, Basel, Switzerland.

Cynthia Bézier

14-10-2022 | Neurology | Video

Novel MAS receptor activator shows potential for SMA

Cynthia Bézier discusses findings from a preclinical study showing that BIO101 – a novel orally administered compound that activates the MAS receptor – may represent a promising treatment option for spinal muscular atrophy.

Funding for independent interviews at WMS 2022 was provided by F. Hoffmann-La Roche Ltd, Basel, Switzerland.

Martin Cheung

14-10-2022 | Neurology | Video

DLC1 isoform 1 may represent a new target for SMA gene therapy

Martin Cheung describes a genetic study showing that loss or reduced expression of DLC1 isoform 1 may contribute to motor neuron defects in spinal muscular atrophy, and discusses the potential of this gene as a therapeutic target.

Funding for independent interviews at WMS 2022 was provided by F. Hoffmann-La Roche Ltd, Basel, Switzerland.