Behandlung von SMA bei Jugendlichen und Erwachsenen


Dear SMA Community,
Following your request for an update on new data in adult patients, we wanted to share the information on the new real-world study of nusinersen use in teens and adults with SMA published in The Lancet Neurology on March 18th, in a manuscript titled "Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study”.

Results from this independent study conducted in Germany by Hagenacker T, et al. demonstrates the safety and efficacy of SPINRAZA (nusinersen) in teens and adults with 5q spinal muscular atrophy (SMA). The study examined the largest adult patient cohort to date and adds to the growing body of evidence supporting SPINRAZA treatment in adults with SMA. Results show clinically meaningful improvements in motor function.

The study contains data from 124 adult SMA patients (mainly, types 2 and 3) from ten neuromuscular treatment centers in Germany. The results are based on the evaluation of several motor function scales, including the HFMSE (Hammersmith Functional Motor Scale Expanded), RULM (Revised Upper Limb Module) and the 6MWT (6-Minute Walk Test).

This publication is an important milestone for the SMA community, to further inform teen and adult treatment decisions with the goal of improving patient outcomes.

Link: Safety and Treatment Effects of Nusinersen in adult SMA