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How did four years of oral treatment affect the health of children with the most severe type of SMA: results from the FIREFISH clinical trial

MedExpress Team

medexpress.pl

Published Oct. 4, 2023 13:34

How did four years of oral treatment affect the health of children with the most severe type of SMA: results from the FIREFISH clinical trial - Header image
The new data, obtained during four years of follow-up, confirm the long-term effectiveness of the of the oral drug (risdiplam) in treating children with the most severe form of spinal muscular atrophy (SMA type 1). That's the data from the FIREFISH clinical trial, which was presented at this year's Cure SMA Congress, held in early July in Orlando, USA. One of the presented patients participating in this study was 5-year-old Zosia from Poland.

The FIREFISH clinical trial was a so-called registration study, which means that based on the positive results obtained in it, the European Medicines Agency (EMA) approved the oral drug risdiplam for the treatment of spinal muscular atrophy. Eligible to participate in this study were infants who were between 1 and 7 months of age at the time of inclusion in the clinical trial. All of these children were diagnosed - using genetic testing - with SMA type 1, the most severe form of spinal muscular atrophy. "The process of death of motoneurons israpid in SMA 1 patients and begins either right after birth or even prenatally. Therefore, children with type 1 SMA manifest symptoms of the disease as early as the first six months of life. Historically, in the natural course of the disease, that is, without the administration of treatment, these children never sat up independently, 90% of them required ventilator support and nutritional support before the age of 1, and many of them died within the first two years of life due to severe respiratory muscle weakness and complications from severe lung infections. SMA was until recently the most common cause of infant death.The fact that we can now treat spinal muscular atrophy and stop the progression of the disease is a great breakthrough and something that makes us very optimistic," says Prof. Maria Mazurkiewicz-Bełdzińska, MD, from the Department of Developmental Neurology at the Medical University of Gdansk, president of the Polish Societ...

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