Multidisciplinary Approach Improves Spinraza Dosing Process in SMA Patients, US Study Shows
Adopting a multidisciplinary approach can optimize pretreatment evaluations and improve the dosing process for Spinraza (nusinersen) in people with spinal…
Articles by Marta Figueiredo, PhD
Targeted Genetic Screening Useful in Diagnosing Patients with SMA-like Symptoms
Targeted next generation sequencing (NGS) — a powerful genetics method that can identify mutations in several genes simultaneously —…
Risdiplam Improves Motor Function in People with SMA Types 2 and 3, Interim Phase 2/3 Data Show
Risdiplam significantly improves motor function in pediatric and young adult patients with spinal muscular atrophy (SMA) types 2 and 3,…
Mutation Newly Linked to Adult-Onset Distal SMA in Spanish Siblings, Case Study Reports
A mutation in the KIF5A gene, found in four Spanish siblings, causes a rare form of spinal muscular atrophy (SMA)…
New Potential SMA Therapeutic Target Found in Muscle Cells, Mouse Study Suggests
Deficits in the secretion of a protein called CTRP3 by muscle cells contribute to the significantly lower survival motor…
SMA Patients May Benefit from Low-fat Diet, Mouse Study Suggests
Adhering to a low-fat diet may be beneficial for people with spinal muscular atrophy (SMA), a mouse study suggests.
SMN Protein Study in Fetuses and Children Emphasizes Importance of Early SMA Treatment, Newborn Screening
Levels of the survival motor neuron (SMN) protein — which are significantly low in spinal muscular atrophy (SMA) patients —…
Spinraza Improves or Stabilizes Motor, Lung Function in SMA Type 3 Adults, German Study Suggests
Spinraza (nusinersen) improves or stabilizes motor and lung functions in adults with spinal muscular atrophy (SMA) type 3, an…
Risdiplam Continues to Show Survival, Motor Function Improvements, According to Trial Results
Risdiplam continues to show promise as a safe and effective therapy for people with spinal muscular atrophy (SMA) types 1,…
Researchers Develop Fast, Low-Cost, and Non-invasive Prenatal Screening Test for SMA
Chinese researchers have developed a fast, low-cost, and non-invasive maternal blood test for prenatal screening of spinal muscular atrophy…