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Page last updated: 27th March 2020


Dr James Sleigh, our Scientific Research Correspondent, reported on a study published in the Lancet this month, which showed nusinersen to be safe and effective for the treatment of SMA in adults:

Dr. Hagenacker and team carried out an observational study of 173 adults with SMN-linked SMA at ten academic clinical sites in Germany.

Patients included in the study had SMA Types 1, 2 or 3, and were aged between 16 and 65 years old when first treated. Nusinersen was injected into the fluid bathing the brain and spinal cord (“intrathecally”), in accordance with the label, for a minimum treatment time of 6 months to an end follow-up of a maximum of 14 months.

The primary outcome was the change in motor function score of a standard test used for assessing how SMA is progressing called the Hammersmith Functional Motor Scale-Expanded (HFMSE). Motor function was assessed after 6, 10 and 14 months of treatment and compared with pre-treatment scores (i.e. baseline scores).

Motor function scores were significantly improved compared with baseline at 6 months,

10 months and 14 months. Clinically meaningful improvements (at least 3 points increase) in HFMSE scores were seen in 35 (28%) of 124 patients at 6 months, 33 (35%) of 92 at 10 months, and 23 (40%) of 57 at 14 months.

This suggests that nusinersen treatment is effective at combating the impact of SMA in adults in at least some patients.

The most frequent adverse effects in the 173 patients were headache (61 [35%] patients), back pain (38 [22%]), and nausea (19 [11%]). However, no serious adverse events were reported.

The main limitation of the study is that it was observational in design, meaning that the effect of nusinersen treatment could not be compared with patients in a non-treated control group. Proving effectiveness of a drug is tricky when no control group is present, especially in a disease that can progress quite slowly in adulthood.

Nevertheless, the data presented in the Lancet provide good evidence for the safety and efficacy of nusinersen in the treatment of adults with SMN-linked (5q) SMA, with many patients showing clinically meaningful improvements in muscle function.


Further information

Abstract of The Lancet Publication >

About Nusinersen >