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The RESPOND study, an open-label trial, evaluates the clinical outcomes and safety of SPINRAZA over a two-year period. The recent findings presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference suggest that SPINRAZA may further benefit SMA patients who have undergone gene therapy.
In the study, participants with two copies of the SMN2 gene, all of whom had elevated baseline NfL levels, showed a mean decrease of 70% for infants and 78% for children older than 9 months. For the small group with three SMN2 copies, those with elevated baseline levels also experienced reductions, while levels remained stable in one participant without elevated baseline levels.
These biomarker results complement previous findings from June 2023, which reported improvements in motor function in most participants. No new safety concerns were identified, and no serious adverse events were considered related to SPINRAZA or led to study withdrawal.
SPINRAZA, approved in over 71 countries, is a foundational treatment for SMA, with more than 14,000 individuals treated globally. It is an antisense oligonucleotide that targets the root cause of SMA by increasing the production of full-length survival motor neuron (SMN) protein. The drug is administered directly into the central nervous system and has demonstrated sustained efficacy and a well-established safety profile.
Biogen, founded in 1978, is a biotechnology leader focusing on innovative science to deliver transformative medicines. The company has licensed global rights to SPINRAZA from Ionis Pharmaceuticals, Inc. (NASDAQ:IONS).
The information in this article is based on a press release statement.
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