Raising Levels of Alpha-COP Protein Seen to Ease Disease Severity in Mouse Model
Elevated levels of the protein alpha-COP, involved in cellular transport and self-cleansing processes, extended survival in a mouse model of…
Articles by Ana Pena, PhD
NICE Recommends Funding of Spinraza in England’s National Health System for all SMA Patients
Patients of all ages with spinal muscular atrophy (SMA) will now…
High-intensity Training Improves Fitness While Being Safe for Patients with Kennedy’s Disease, Small Study Suggests
High-intensity training can improve fitness and may be performed safely by patients with spinal and bulbar muscular atrophy (SBMA), a small…
Survival Rate is Up for Adults and Children with Neuromuscular Disorders, Canadian Study Reveals
Adults and children with neuromuscular disorders — including those with spinal muscular atrophy (SMA) — are living longer, according to a…
Saudi Arabian Doctors Need to be More Aware of Guidelines and New Care Options for SMA, Study Says
Saudi Arabian doctors need to be more aware of consensus guidelines for managing patients with spinal muscular atrophy (SMA) and their…
Skin Lesions of Infant with SMA1 Cured After Spinraza Treatment, Case Study Reports
Treatment with Spinraza (nusinersen) was able to resolve many severe…
Perceived Fatigue Not Linked to Physical Factors, Quality of Life in SMA Patients, Study Reports
The sense of fatigue perceived by patients with spinal muscular atrophy…
LMI070 Oral Candidate For SMA Type 1 Holds Promise, Preclinical Study Shows
LMI070 (branaplam), a candidate oral therapy under development by Novartis, is being tested in a Phase 1/2 clinical trial…
3-D Cell Model Mimics SMA Motor Neurons, is Good Platform For Testing Possible Therapies, Study Suggests
A 3-D cell model derived from patients’ stem cells is able to mimic motor neurons from…
Simple Hand Exercise Helps Detect Fatigability in SMA Type 2 Patients, Study Shows
Patients with spinal muscular atrophy (SMA) are more susceptible to becoming tired during physical activity, which, in addition…