Study: Drug Therapy Improving Function in Kids with SMA Types 2 & 3

A newly published study says a drug therapy for children with spinal muscular atrophy (SMA) is improving their upper-extremity function.

The study, published in Orphanet Journal of Rare Diseases in October, is called, “Improved Upper Limb Function in Non-Ambulant Children with SMA Type 2 and 3 During Nusinersen Treatment: A Prospective 3-Years SMArtCARE Registry Study.”

The SMArtCARE Registry collects data from SMA patients in Germany, Austria, and Switzerland.

The study centers on SMArtCARE registry data for children under age 18 with SMA Type 2 or 3 who have taken Nusinersen — marketed as Spinraza — and were under a 38-month follow-up period.

Spinraza is taken three times a year after four initial doses, according to its manufacturer.

Researchers studied 256 non-ambulatory pediatric patients. “Improvements in motor function were more prominent in upper limb: 32.4 percent of patients experienced clinically meaningful improvements in RULM [Revised Upper Limb Module, an assessment of upper-extremity function in patients with SMA], and 24.6 percent in the HFMSE [Hammersmith Functional Motor Scale Expanded, which assesses high-functioning SMA Type 2 and 3 patients].”

The study said 4.3 percent and 1.2 percent of patients had worsening results in HFMSE or RULM scores, respectively.

“Our results demonstrate clinically meaningful improvements or stabilization of disease progression in non-ambulant, pediatric patients with SMA under Nusinersen treatment,” the study said. “Changes were most evident in upper-limb function and were observed continuously over the follow-up period. Our data confirm clinical trial data, while providing longer follow-up, an increased number of treated patients, and a wider range of age and disease severity.”


About the Author

Laurie Watanabe is the editor of Mobility Management. She can be reached at [email protected].

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