CHIT1 Immune Protein May Be Marker of Response to Spinraza
Levels of an immune protein called CHIT1 change over time in children with spinal muscular atrophy (SMA) who undergo…
Articles by Marisa Wexler, MS
Evrysdi Found to Help Previously Treated SMA Patients in Trial
Treatment with Evrysdi (risdiplam) increased levels of SMN protein and stabilized motor function in a Phase 2 clinical trial…
New Study Suggests Zolgensma Could Work in Adults With SMA
The gene therapy Zolgensma (onasemnogene abeparvovec-xioi), approved in the U.S. for very young children with spinal muscular atrophy…
Experts Share Insights on Deciding Treatment in Changing Landscape
Doctors need to be well-educated about options in the evolving treatment landscape to help patients with spinal muscular atrophy…
Majority in SMA Community Survey Support Prenatal Treatment: Study
Note: This story was updated Sept. 19, 2022, to correct that 78.9% of the survey respondents would choose an…
CADTH: Spinraza Should Not Be Funded for Adults in Canada
In its final decision, the Canadian Agency for Drugs and Technologies in Health (CADTH) recommended against Spinraza (nusinersen)…
Zolgensma Label to Include Warning of Acute Liver Failure Deaths
Prescribing information for Zolgensma (onasemnogene abeparvovec-xioi), an approved gene therapy for spinal muscular atrophy (SMA), is being updated…
Support Urged for Parents Informing Their Children of SMA Diagnosis
Children diagnosed with spinal muscular atrophy (SMA) are usually informed about the diagnosis by their parents, who frequently lack…
Elevated GFAP Levels Linked to More Motor Impairment in SMA
Higher levels of the glial fibrillary acidic protein (GFAP) in the fluid around the brain and spinal cord are associated…
Newborn Screening Allows Timely SMA Treatment of Baby in Japan
In the first year of a newborn screening program for spinal muscular atrophy (SMA) in Japan, more than 10,000…