This part of the website contains news related to SMA, such as meeting reports and newsletters.

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LIS-N & LER-N report 2019

This report gives insight in the Listen & Learn meeting we had in 2019. The report gives insight in the speakers and what has been discussed throughout that meeting.

LIS-N & LER-N report 2020

This report gives insight in the Listen & Learn meeting we had in 2020. The report gives insight in the speakers and what has been discussed throughout that meeting.

SMA Newsletter Norway 2019

We sent out a newsletter to some of our stakeholders in 2019. You can find a copy here.

SMA Newsletter Norway 2020

We sent out a newsletter to some of our stakeholders in May 2020. In this newsletter you will find topics, all related to the Listen & Learn meeting that was held in December 2019. You can find a copy here.

SMA newsletter adult data 2021

This newsletter provides insight in the real-world data regarding adult SMA treatment. Dr. Tim Hagenacker and Prof. Christoph Kleinschnitz share their experiences via videos.

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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 (01/2021) for further details.