NURTURE study participants also achieved a mean CHOP INTEND score, which measures general motor function among infants with SMA, of 58.4 at last visit (out of a maximum score of 64). Many continue to improve and maintain these scores beyond a point in time at which untreated individuals with Type 1 SMA would experience a significant decline. Overall, the study showed that SPINRAZA was well-tolerated and no new safety concerns were identified.
Also presented at the MDA Clinical Conference was a case series, “Nusinersen Experience in Teenagers and Young Adults With Spinal Muscular Atrophy,” which showcased SPINRAZA’s stabilizing or improving effect on teens and young adults with Type 2 or 3 SMA.
In the case series, participants (n=5) were 14 to 15 years old at the start of Study CS2, and 17 to 19 years old at the time of their last visit in the extension Study CS12. One participant was Type 2 and four were Type 3, and all received multiple doses of SPINRAZA over 2.5 years of observation. The results included improvement on the Hammersmith Functional Motor Scale–Expanded (HFMSE); stabilization on the Upper Limb Module (ULM); improvement in the Six-Minute Walk Test (6MWT); and stable or improved scores on the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND).
“The case series demonstrated SPINRAZA’s effectiveness in teens and young adults with SMA Type 2 or 3,” said Dr. John Day, M.D., Ph.D., lead study author, Stanford University Medical Center, California. “The study participants demonstrated stable or slightly improved motor function and quality of life during two years of treatment – and even afterward, we have continued to see improved and maintained stability and motor benefits with SPINRAZA. This differs importantly from untreated teens and young adults with SMA, who experience a decline in motor function, specifically in reduced walking distance and upper limb activities, as well as in health-related quality of life.