This part of the website provides insights on management of SMA across patient populations
Agneta Markstrom presentation
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Respiratory support with mobilization of secretions and use of non-invasive ventilation play an important role in the management of children with SMA, to prevent chest infections and promote thoracic development and compliance. Agneta Markstrom presented an overview of respiratory care during our LISN and LERN meeting in 2019.
Zac's Playday video
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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.
Marika Pane interview
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This interview was held with Marika Pane during the Listen & Learn meeting in December 2019. She explains more about her role and the study in which she was involved, including results.
Thomas Sejersen interview
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This interview was held with Thomas Sejersen during the Listen & Learn meeting in December 2019. He provides more insight in the programme for 2019's Listen & Learn meeting.
Agneta Markstrom interview
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This interview was held with Agneta Markström during the Listen & Learn meeting in December 2019. She describes the importance of pulmonary care. A full length recording of her lecture can also be found on this page.
Mar Tulinius interview
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This interview was held with Mar Tulinius during the Listen & Learn meeting in December 2019. He explains more about his role in some of the trials, also related to newborn screening.
Early Signs brochure
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This brochure shows insight in how early signs of SMA can be recognized at an early stage.
Lumbar Puncture Brochure
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A brochure which describes the process of a lumbar puncture for those patients who are to undergo that procedure.
How is Spinraza working in adults?
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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”.
PubExplainer: A critical review and meta-analysis (Coratti et al., 2021)
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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.
PubExplainer: Nusinersen safety and efficacy in adult SMA patients (Maggi et al., 2020)
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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).
Hagenacker pubexplainer
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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.
Video Interview Prof. Dr. Hagenacker "New Data on the Treatment of adult SMA with NUS"
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This video shows an expert interview with Dr. Tim Hagenacker, Chief Senior Physician of the Clinic for Neurology, Essen University Hospital.
Video Interview Prof. Dr. Kleinschnitz "Treatment of SMA in adult patients"
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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.
Presentation Habilitation and rehabilitation in spinal muscular atrophy
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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.
Outcome measures - current use and future needs, AK Kroksmark
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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.
Presentation Transition from adolescents into adult department
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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.
CHOP-INTEND video instructions
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CHOP-INTEND - Instructions on how to perform the CHOP-INTEND
REFERENCES
Precautions
Patients with profound hypotonia and respiratory failure at birth, a setting in which Spinraza has not been studied, may not experience a clinically meaningful benefit due to severe SMN protein deficiency. There is a risk of adverse reactions occurring as part of the lumbar puncture (e.g headache, back pain, vomiting; see section 4.8 in the SPC. Potential difficulties with the route of administration may be seen in very young patients and those with scoliosis. The use of ultrasound or other imaging techniques to assist with intrathecal administration of Spinraza can be considered at the physicians discretion.
Renal toxicity has been observed after administration of other subcutaneously and intravenously administered antisense nucleotides. If clinically indicated, urine protein testing (preferably using a first morning urine specimen) is recommended. For persistent elevated urinary protein, further evaluation should be considered.
Thrombocytopenia, coagulation abnormalities, including acute severe thrombocytopenia and renal toxicity have been observed after administration of other subcutaneously or intravenously administered antisense oligonucleotides. If clinically indicated, laboratory testing of platelet, coagulation and urine protein is recommended prior to administration of Spinraza.
There have been reports of communicating hydrocephalus not related to meningitis or bleeding in patients treated with nusinersen in the post-marketing setting. See section 4.4 in the SPC (01/2022) for further details.