by Marta Figueiredo PhD | Jun 17, 2022 | News
Pediatric neurologists’ expectations in terms of treatment benefits are decidedly higher for infants and toddlers with spinal muscular atrophy (SMA) types 1 and 2 than for adolescent patients, a study in Spain shows. Notably, neurologists with low aversion to...
by Marta Figueiredo PhD | Jun 3, 2022 | News
Infants with spinal muscular atrophy (SMA) who are being screened for Zolgensma may present with maternal antibodies against the therapy’s viral carrier — making them ineligible for the therapy — until the age of 8 months, a small study shows. This means that an...
by Marta Figueiredo PhD | May 20, 2022 | News
Evrysdi (risdiplam) was generally safe and well tolerated in a broader group of children and adults with spinal muscular atrophy (SMA) types 1 and 2 than that included in clinical trials supporting the therapy’s approval. These were the findings of an analysis of...
by Marta Figueiredo PhD | May 18, 2022 | News
Zolgensma appears generally safe and well tolerated among spinal muscular atrophy (SMA) patients weighing 8.5 kilograms (about 18.7 lbs) or more, with no new safety concerns identified in the heavier group. Notably, 8.5 kilograms was the maximum weight of SMA patients...
by Marta Figueiredo PhD | Apr 29, 2022 | News
Treatment with Spinraza (nusinersen) appears safe in infants and children with spinal muscular atrophy (SMA) who responded poorly to the gene therapy Zolgensma, early safety data from the first nine RESPOND trial patients show. Motor and lung function were the most...