Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of…
Articles by Marta Figueiredo, PhD
Spinraza Improves Motor Function in Case of Teenager with Late-onset SMA
Fourteen months of treatment with Spinraza (nusinersen) resulted in improved motor function in a previously untreated 15-year-old boy with late-onset…
EMBRACE Trial Supports Spinraza’s Benefits in Broad Range of SMA Patients
Treatment with Spinraza (nusinersen) was found to reduce the use of ventilation support and to improve motor function in…
Better Ways of Capturing Progression in Types 2 and 3 Identified in Study
Four measures of motor ability — including one of continuous upper limb activity that can be done at the home…
Top 10 SMA Stories of 2020
SMA News Today brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to spinal…
Zolgensma Linked in 3 Cases to Serious But Treatable Blood Disorder
Some children with spinal muscular atrophy (SMA) may develop thrombotic microangiopathy (TMA) —…
Zolgensma-associated Liver Abnormalities Common but Manageable, Study Shows
Most children with spinal muscular atrophy (SMA) show an abnormal increase in the levels of liver enzymes — suggestive of liver damage…
Spinraza Linked to Temporary Abnormalities in Immune Cells in 2 SMA Infants
Repeated administrations of Spinraza (nusinersen) were associated with the presence of macrophages — immune cells specialized in recognizing and…
Extensive Trial Monitoring Confirms Evrysdi Does Not Damage Vision
Evrysdi (risdiplam) at its approved and therapeutic dose does not lead to eye damage in children or adults with spinal…
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with…